-
1
-
-
67649228313
-
Genetics. The promise of a cure: 20 years and counting
-
Couzin-Frankel J (2009) Genetics. The promise of a cure: 20 years and counting. Science 324:1504-1507
-
(2009)
Science
, vol.324
, pp. 1504-1507
-
-
Couzin-Frankel, J.1
-
2
-
-
0029147628
-
Nonviral gene therapy: The promise of genes as pharmaceutical products
-
Ledley FD (1995) Nonviral gene therapy: the promise of genes as pharmaceutical products. Hum Gene Ther 6:1129-1144
-
(1995)
Hum Gene Ther
, vol.6
, pp. 1129-1144
-
-
Ledley, F.D.1
-
3
-
-
0026718123
-
Human gene therapy comes of age
-
Miller DA (1992) Human gene therapy comes of age. Nature 357:455-460
-
(1992)
Nature
, vol.357
, pp. 455-460
-
-
Miller, D.A.1
-
4
-
-
67650498100
-
Human genetics: One gene, twenty years
-
Pearson H (2009) Human genetics: one gene, twenty years. Nature 460:164-169
-
(2009)
Nature
, vol.460
, pp. 164-169
-
-
Pearson, H.1
-
5
-
-
0029799638
-
Gene therapy in the United States: A five-year status report
-
Ross G, Erickson R, Knorr D et al (1996) Gene therapy in the United States: a five-year status report. Hum Gene Ther 7:1781-1790
-
(1996)
Hum Gene Ther
, vol.7
, pp. 1781-1790
-
-
Ross, G.1
Erickson, R.2
Knorr, D.3
-
6
-
-
0038745599
-
Progress and problems with the use of viral vectors for gene therapy
-
Thomas CE, Ehrhardt A, Kay MA (2003) Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet 4:346-358
-
(2003)
Nat Rev Genet
, vol.4
, pp. 346-358
-
-
Thomas, C.E.1
Ehrhardt, A.2
Kay, M.A.3
-
7
-
-
38949104082
-
How not to be seen: Immune-evasion strategies in gene therapy
-
Zaldumbide A, Hoeben RC (2007) How not to be seen: immune-evasion strategies in gene therapy. Gene Ther 15:239-246
-
(2007)
Gene Ther
, vol.15
, pp. 239-246
-
-
Zaldumbide, A.1
Hoeben, R.C.2
-
8
-
-
0034581163
-
Tet repressor-based system for regulated gene expression in eukaryotic cells: Principles and advances
-
Baron U, Bujard H (2000) Tet repressor-based system for regulated gene expression in eukaryo - tic cells: principles and advances. Methods Enzymol 327:401-421
-
(2000)
Methods Enzymol
, vol.327
, pp. 401-421
-
-
Baron, U.1
Bujard, H.2
-
9
-
-
67651087321
-
Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications
-
Brown BD, Naldini L (2009) Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications. Nat Rev Genet 10:578-585
-
(2009)
Nat Rev Genet
, vol.10
, pp. 578-585
-
-
Brown, B.D.1
Naldini, L.2
-
10
-
-
0031197176
-
Controlling mammalian gene expression with small molecules
-
Clackson T (1997) Controlling mammalian gene expression with small molecules. Curr Opin Chem Biol 1:210-218
-
(1997)
Curr Opin Chem Biol
, vol.1
, pp. 210-218
-
-
Clackson, T.1
-
11
-
-
52649177737
-
The triple helix: 50 years later, the outcome
-
Duca M, Vekhoff P, Oussedik K et al (2008) The triple helix: 50 years later, the outcome. Nucleic Acids Res 36:5123-5138
-
(2008)
Nucleic Acids Res
, vol.36
, pp. 5123-5138
-
-
Duca, M.1
Vekhoff, P.2
Oussedik, K.3
-
12
-
-
50549093286
-
Gene transfer: The challenge of regulated gene expression
-
Guo ZS, Li Q, Bartlett DL, Yang J Y, Fang B (2008) Gene transfer: the challenge of regulated gene expression. Trends Mol Med 14:410-418
-
(2008)
Trends Mol Med
, vol.14
, pp. 410-418
-
-
Guo, Z.S.1
Li, Q.2
Bartlett, D.L.3
Yang, J.Y.4
Fang, B.5
-
13
-
-
0032134253
-
Inducible control of gene expression: Prospects for gene therapy
-
Harvey DM, Caskey CT (1998) Inducible control of gene expression: prospects for gene therapy. Curr Opin Chem Biol 2:512-518
-
(1998)
Curr Opin Chem Biol
, vol.2
, pp. 512-518
-
-
Harvey, D.M.1
Caskey, C.T.2
-
14
-
-
0141681221
-
Intracellular antibodies and challenges facing their use as therapeutic agents
-
Lobato MN, Rabbitts TH (2003) Intracellular antibodies and challenges facing their use as therapeutic agents. Trends Mol Med 9:390-396
-
(2003)
Trends Mol Med
, vol.9
, pp. 390-396
-
-
Lobato, M.N.1
Rabbitts, T.H.2
-
15
-
-
50049095016
-
Drug-inducible and simultaneous regulation of endogenous genes by single-chain nuclear receptor-based zinc-finger transcription factor gene switches
-
Magnenat L, Schwimmer LJ, Barbas CF (2008) Drug-inducible and simultaneous regulation of endogenous genes by single-chain nuclear receptor-based zinc-finger transcription factor gene switches. Gene Ther 15:1223-1232
-
(2008)
Gene Ther
, vol.15
, pp. 1223-1232
-
-
Magnenat, L.1
Schwimmer, L.J.2
Barbas, C.F.3
-
16
-
-
0035312546
-
Recognition of antigens by single-domain antibody fragments: The superfluous luxury of paired domains
-
Muyldermans S, Cambillau C, Wyns L (2001) Recognition of antigens by single-domain antibody fragments: the superfluous luxury of paired domains. Trends Biochem Sci 26:230-235
-
(2001)
Trends Biochem Sci
, vol.26
, pp. 230-235
-
-
Muyldermans, S.1
Cambillau, C.2
Wyns, L.3
-
17
-
-
1942537842
-
Gene therapy progress and prospects: Transcription regulatory systems
-
Toniatti C, Bujard H, Cortese R, Ciliberto G (2004) Gene therapy progress and prospects: transcription regulatory systems. Gene Ther 11:649-657
-
(2004)
Gene Ther
, vol.11
, pp. 649-657
-
-
Toniatti, C.1
Bujard, H.2
Cortese, R.3
Ciliberto, G.4
-
18
-
-
0029883563
-
Gene transfer to augment the therapeutic index of anticancer chemotherapy
-
Baum C, Margison GP, Eckert H-G et al (1996) Gene transfer to augment the therapeutic index of anticancer chemotherapy. Gene Ther 3:1-3
-
(1996)
Gene Ther
, vol.3
, pp. 1-3
-
-
Baum, C.1
Margison, G.P.2
Eckert, H.-G.3
-
19
-
-
36849060584
-
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state
-
Brown BD, Gentner B, Cantore A et al (2007) Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nat Biotechnol 25:1457-1467
-
(2007)
Nat Biotechnol
, vol.25
, pp. 1457-1467
-
-
Brown, B.D.1
Gentner, B.2
Cantore, A.3
-
20
-
-
0026720075
-
Tight control of gene expression in mammalian cells by tetracyclineresponsive promoters
-
Gossen M, Bujard H (1992) Tight control of gene expression in mammalian cells by tetracyclineresponsive promoters. Proc Natl Acad Sci USA 89:5547-5551
-
(1992)
Proc Natl Acad Sci USA
, vol.89
, pp. 5547-5551
-
-
Gossen, M.1
Bujard, H.2
-
21
-
-
0141681221
-
Intracellular antibodies and challenges facing their use as therapeutic agents
-
Lobato MN, Rabbitts TH (2003) Intracellular antibodies and challenges facing their use as therapeutic agents. Trends Mol Med 9:390-396
-
(2003)
Trends Mol Med
, vol.9
, pp. 390-396
-
-
Lobato, M.N.1
Rabbitts, T.H.2
-
22
-
-
4644353198
-
Comparative analysis of inducible expression systems in transient transfection studies
-
Meyer-Ficca ML, Meyer RG, Kaiser H et al (2004) Comparative analysis of inducible expression systems in transient transfection studies. Anal Biochem 334:9-19
-
(2004)
Anal Biochem
, vol.334
, pp. 9-19
-
-
Meyer-Ficca, M.L.1
Meyer, R.G.2
Kaiser, H.3
-
23
-
-
0029865151
-
Ecdysone-inducible gene expression in mammalian cells and transgenic mice
-
No D, Yao TP, Evans RM (1996) Ecdysone-inducible gene expression in mammalian cells and transgenic mice. Proc Natl Acad Sci USA 93:3346-3351
-
(1996)
Proc Natl Acad Sci USA
, vol.93
, pp. 3346-3351
-
-
No, D.1
Yao, T.P.2
Evans, R.M.3
-
24
-
-
33744810303
-
Pharmacologic transgene control systems for gene therapy
-
Weber W, Fussenegger M (2006) Pharmacologic transgene control systems for gene therapy. J Gene Med 8:535-556
-
(2006)
J Gene Med
, vol.8
, pp. 535-556
-
-
Weber, W.1
Fussenegger, M.2
-
25
-
-
67651087321
-
Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications
-
Brown BD, Naldini L (2009) Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications. Nat Rev Genet 10:578-585
-
(2009)
Nat Rev Genet
, vol.10
, pp. 578-585
-
-
Brown, B.D.1
Naldini, L.2
-
26
-
-
60149088848
-
Origins and mechanisms of miRNAs and siRNAs
-
Carthew RW, Sontheimer EJ (2009) Origins and mechanisms of miRNAs and siRNAs. Cell 136:642-655
-
(2009)
Cell
, vol.136
, pp. 642-655
-
-
Carthew, R.W.1
Sontheimer, E.J.2
-
27
-
-
58749104364
-
The promises and pitfalls of RNA-interference-based therapeutics
-
Castanotto D, Rossi JJ (2009) The promises and pitfalls of RNA-interference-based therapeutics. Nature 457:426-433
-
(2009)
Nature
, vol.457
, pp. 426-433
-
-
Castanotto, D.1
Rossi, J.J.2
-
28
-
-
52649177737
-
The triple helix: 50 years later, the outcome
-
Duca M, Vekhoff P, Oussedik K et al (2008) The triple helix: 50 years later, the outcome. Nucleic Acids Res 36:5123-5138
-
(2008)
Nucleic Acids Res
, vol.36
, pp. 5123-5138
-
-
Duca, M.1
Vekhoff, P.2
Oussedik, K.3
-
29
-
-
33645148088
-
The silent treatment: Sirnas as small molecule drugs
-
Dykxhoorn DM, Palliser D, Lieberman J (2006) The silent treatment: siRNAs as small molecule drugs. Gene Ther 13:541-552
-
(2006)
Gene Ther
, vol.13
, pp. 541-552
-
-
Dykxhoorn, D.M.1
Palliser, D.2
Lieberman, J.3
-
30
-
-
33750991861
-
The potential of oligonucleotides for therapeutic applications
-
Fichou Y, Férec C (2006) The potential of oligonucleotides for therapeutic applications. Trends Biotechnol 24:563-570
-
(2006)
Trends Biotechnol
, vol.24
, pp. 563-570
-
-
Fichou, Y.1
Férec, C.2
-
31
-
-
0037062951
-
RNA interference
-
Hannon GJ (2002) RNA interference. Nature 418:244-251
-
(2002)
Nature
, vol.418
, pp. 244-251
-
-
Hannon, G.J.1
-
32
-
-
0036636074
-
Nucleic-acid therapeutics: Basic principles and recent applications
-
Opalinska JB, Gewirtz AM (2002) Nucleic-acid therapeutics: basic principles and recent applications. Nat Rev Drug Discov 1:503-514
-
(2002)
Nat Rev Drug Discov
, vol.1
, pp. 503-514
-
-
Opalinska, J.B.1
Gewirtz, A.M.2
-
33
-
-
33846914204
-
Gene therapy progress and prospects: RNA aptamers
-
Que-Gewirth NS, Sullenger BA (2007) Gene therapy progress and prospects: RNA aptamers. Gene Ther 14:283-291
-
(2007)
Gene Ther
, vol.14
, pp. 283-291
-
-
Que-Gewirth, N.S.1
Sullenger, B.A.2
-
36
-
-
0037213559
-
Mammalian RNAi for the masses
-
Shi Y (2003) Mammalian RNAi for the masses. Trends Genet 19:9-12
-
(2003)
Trends Genet
, vol.19
, pp. 9-12
-
-
Shi, Y.1
-
37
-
-
24144458271
-
Ribozymes, DNAzymes and small interfering RNAs as therapeutics
-
Sioud M, Iversen PO (2005) Ribozymes, DNAzymes and small interfering RNAs as therapeutics. Curr Drug Targets 6:647-653
-
(2005)
Curr Drug Targets
, vol.6
, pp. 647-653
-
-
Sioud, M.1
Iversen, P.O.2
-
38
-
-
6044248871
-
Therapeutic potential of RNA interference
-
Stevenson M (2004) Therapeutic potential of RNA interference. N Engl J Med 351:1772-1777
-
(2004)
N Engl J Med
, vol.351
, pp. 1772-1777
-
-
Stevenson, M.1
-
39
-
-
0036245743
-
Expanding small RNA interference
-
Tuschl T (2002) Expanding small RNA interference. Nat Biotechnol 20:446-448
-
(2002)
Nat Biotechnol
, vol.20
, pp. 446-448
-
-
Tuschl, T.1
-
40
-
-
0028019410
-
Gene inhibition using antisense oligodeoxynucleotides
-
Wagner RW (1994) Gene inhibition using antisense oligodeoxynucleotides. Nature 372:333-335
-
(1994)
Nature
, vol.372
, pp. 333-335
-
-
Wagner, R.W.1
-
41
-
-
0242317385
-
Small RNA: Can RNA interference be exploited for therapy?
-
Wall NR, Shi Y (2003) Small RNA: can RNA interference be exploited for therapy? Lancet 362:1401-1403
-
(2003)
Lancet
, vol.362
, pp. 1401-1403
-
-
Wall, N.R.1
Shi, Y.2
-
42
-
-
4344638117
-
In vivo transfer and expression of genes coding for short interfering RNAs
-
Zentilin L, Giacca M (2004) In vivo transfer and expression of genes coding for short interfering RNAs. Curr Pharm Biotechnol 5:341-347
-
(2004)
Curr Pharm Biotechnol
, vol.5
, pp. 341-347
-
-
Zentilin, L.1
Giacca, M.2
-
43
-
-
12644252947
-
The expression cassette determines the functional activity of ribozymes in mammalian cells by controlling their intracellular localization
-
Bertrand E, Castanotto D, Zhou C et al (1997) The expression cassette determines the functional activity of ribozymes in mammalian cells by controlling their intracellular localization. RNA 3:75-88
-
(1997)
RNA
, vol.3
, pp. 75-88
-
-
Bertrand, E.1
Castanotto, D.2
Zhou, C.3
-
44
-
-
0037134020
-
A system for stable expression of short interfering RNAs in mammalian cells
-
Brummelkamp TR, Bernards R, Agami R (2002) A system for stable expression of short interfering RNAs in mammalian cells. Science 296:550-553
-
(2002)
Science
, vol.296
, pp. 550-553
-
-
Brummelkamp, T.R.1
Bernards, R.2
Agami, R.3
-
45
-
-
39549117983
-
Delivery vehicles for small interfering RNA in vivo
-
De Fougerolles AR (2008) Delivery vehicles for small interfering RNA in vivo. Hum Gene Ther 19:125-132
-
(2008)
Hum Gene Ther
, vol.19
, pp. 125-132
-
-
De Fougerolles, A.R.1
-
47
-
-
0034708334
-
Structure and function of the hairpin ribozyme
-
Fedor MJ (2000) Structure and function of the hairpin ribozyme. J Mol Biol 297:269-291
-
(2000)
J Mol Biol
, vol.297
, pp. 269-291
-
-
Fedor, M.J.1
-
49
-
-
8044253998
-
Expression of small, therapeutic RNAs in human cell nuclei
-
Good PD, Krikos AJ, Li SX et al (1997) Expression of small, therapeutic RNAs in human cell nuclei. Gene Ther 4:45-54
-
(1997)
Gene Ther
, vol.4
, pp. 45-54
-
-
Good, P.D.1
Krikos, A.J.2
Li, S.X.3
-
50
-
-
42249093319
-
LNA-mediated microRNA silencing in non-human primates
-
Elmen J, Lindow M, Schutz S et al (2008) LNA-mediated microRNA silencing in non-human primates. Nature 452:896-899
-
(2008)
Nature
, vol.452
, pp. 896-899
-
-
Elmen, J.1
Lindow, M.2
Schutz, S.3
-
52
-
-
28444469246
-
Silencing of microRNAs in vivo with 'antagomirs'
-
Krutzfeldt J, Rajewsky N, Braich R et al (2005) Silencing of microRNAs in vivo with 'antagomirs'. Nature 438:685-689
-
(2005)
Nature
, vol.438
, pp. 685-689
-
-
Krutzfeldt, J.1
Rajewsky, N.2
Braich, R.3
-
55
-
-
85047695997
-
Purging of chronic myelogenous leukemia cells by retrovirally expressed anti-bcr/abl ribozymes with specific celluar compartmentalization
-
Mendoza-Maldonado R, Zentilin L, Giacca M (2001) Purging of chronic myelogenous leukemia cells by retrovirally expressed anti-bcr/abl ribozymes with specific celluar compartmentalization. Cancer Gene Ther 9:71-86
-
(2001)
Cancer Gene Ther
, vol.9
, pp. 71-86
-
-
Mendoza-Maldonado, R.1
Zentilin, L.2
Giacca, M.3
-
56
-
-
65249156118
-
MicroRNAs as a therapeutic target for cardiovascular disease
-
Mishra PK, Tyagi N, Kumar M, Tyagi SC (2009) MicroRNAs as a therapeutic target for cardiovascular disease. J Cell Mol Med 13:778-789
-
(2009)
J Cell Mol Med
, vol.13
, pp. 778-789
-
-
Mishra, P.K.1
Tyagi, N.2
Kumar, M.3
Tyagi, S.C.4
-
58
-
-
0030923062
-
Use of adenoviral VAI small RNA as a carrier for cytoplasmic delivery of ribozymes
-
Prislei S, Buonomo SB, Michienzi A, Bozzoni I (1997) Use of adenoviral VAI small RNA as a carrier for cytoplasmic delivery of ribozymes. RNA 3:677-687
-
(1997)
RNA
, vol.3
, pp. 677-687
-
-
Prislei, S.1
Buonomo, S.B.2
Michienzi, A.3
Bozzoni, I.4
-
59
-
-
1542336952
-
Rational siRNA design for RNA interference
-
Reynolds A, Leake D, Boese Q et al (2004) Rational siRNA design for RNA interference. Nat Biotechnol 22:326-330
-
(2004)
Nat Biotechnol
, vol.22
, pp. 326-330
-
-
Reynolds, A.1
Leake, D.2
Boese, Q.3
-
60
-
-
42549096043
-
Expression strategies for short hairpin RNA interference triggers
-
Rossi JJ (2008) Expression strategies for short hairpin RNA interference triggers. Hum Gene Ther 19:313-317
-
(2008)
Hum Gene Ther
, vol.19
, pp. 313-317
-
-
Rossi, J.J.1
-
61
-
-
0348014774
-
Approaches for the sequence-specific knockdown of mRNA
-
Scherer LJ, Rossi JJ (2003) Approaches for the sequence-specific knockdown of mRNA. Nat Biotechnol 21:1457-1465
-
(2003)
Nat Biotechnol
, vol.21
, pp. 1457-1465
-
-
Scherer, L.J.1
Rossi, J.J.2
-
62
-
-
85047692457
-
The potential for gene repair via triple helix formation
-
Seidman MM, Glazer PM (2003) The potential for gene repair via triple helix formation. J Clin Invest 112:487-494
-
(2003)
J Clin Invest
, vol.112
, pp. 487-494
-
-
Seidman, M.M.1
Glazer, P.M.2
-
63
-
-
9144247189
-
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
-
Soutschek J, Akinc A, Bramlage B et al (2004) Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 432:173-178
-
(2004)
Nature
, vol.432
, pp. 173-178
-
-
Soutschek, J.1
Akinc, A.2
Bramlage, B.3
-
64
-
-
0027376439
-
Antisense oligonucleotides as therapeutic agents: Is the bullet really magical?
-
Stein CA, Chen YC (1993) Antisense oligonucleotides as therapeutic agents: Is the bullet really magical? Science 261:1004-1012
-
(1993)
Science
, vol.261
, pp. 1004-1012
-
-
Stein, C.A.1
Chen, Y.C.2
-
65
-
-
61949361268
-
MicroRNAs as novel regulators of angiogenesis
-
Suarez Y, Sessa WC (2009) MicroRNAs as novel regulators of angiogenesis. Circ Res 104:442-454
-
(2009)
Circ Res
, vol.104
, pp. 442-454
-
-
Suarez, Y.1
Sessa, W.C.2
-
67
-
-
21344433919
-
A phase I clinical trial of a ribozyme-based angiogenesis inhibitor targeting vascular endothelial growth factor receptor-1 for patients with refractory solid tumors
-
Weng DE, Masci PA, Radka SF et al (2005) A phase I clinical trial of a ribozyme-based angiogenesis inhibitor targeting vascular endothelial growth factor receptor-1 for patients with refractory solid tumors. Mol Cancer Ther 4:948-955
-
(2005)
Mol Cancer Ther
, vol.4
, pp. 948-955
-
-
Weng, D.E.1
Masci, P.A.2
Radka, S.F.3
-
69
-
-
0037422066
-
Regulated portals of entry into the cell
-
Conner SD, Schmid SL (2003) Regulated portals of entry into the cell. Nature 422:37-44
-
(2003)
Nature
, vol.422
, pp. 37-44
-
-
Conner, S.D.1
Schmid, S.L.2
-
71
-
-
20744450629
-
Delivery into cells: Lessons learned from plant and bacterial toxins
-
Sandvig K, Van Deurs B (2005) Delivery into cells: lessons learned from plant and bacterial toxins. Gene Ther 12:865-872
-
(2005)
Gene Ther
, vol.12
, pp. 865-872
-
-
Sandvig, K.1
Van Deurs, B.2
-
72
-
-
63049113263
-
Defining macropinocytosis
-
Kerr MC, Teasdale RD (2009) Defining macropinocytosis. Traffic 10:364-371
-
(2009)
Traffic
, vol.10
, pp. 364-371
-
-
Kerr, M.C.1
Teasdale, R.D.2
-
73
-
-
24944523300
-
Clathrin-independent endocytosis: New insights into caveolae and non-caveolar lipid raft carriers
-
Kirkham M, Parton RG (2005) Clathrin-independent endocytosis: new insights into caveolae and non-caveolar lipid raft carriers. Biochim Biophys Acta 1745:273-286
-
(2005)
Biochim Biophys Acta
, vol.1745
, pp. 273-286
-
-
Kirkham, M.1
Parton, R.G.2
-
74
-
-
34548231783
-
"Alternative" endocytic mechanisms exploited by pathogens: New avenues for therapeutic delivery?
-
Medina-Kauwe LK (2007) "Alternative" endocytic mechanisms exploited by pathogens: new avenues for therapeutic delivery? Adv Drug Deliv Rev 59:798-809
-
(2007)
Adv Drug Deliv Rev
, vol.59
, pp. 798-809
-
-
Medina-Kauwe, L.K.1
-
75
-
-
0345706824
-
Caveosomes and endocytosis of lipid rafts
-
Nichols B (2003) Caveosomes and endocytosis of lipid rafts. J Cell Sci 116:4707-4714
-
(2003)
J Cell Sci
, vol.116
, pp. 4707-4714
-
-
Nichols, B.1
-
76
-
-
0037134014
-
Local actin polymerization and dynamin recruitment in SV40-induced internalization of caveolae
-
Pelkmans L, Puntener D, Helenius A (2002) Local actin polymerization and dynamin recruitment in SV40-induced internalization of caveolae. Science 296:535-539
-
(2002)
Science
, vol.296
, pp. 535-539
-
-
Pelkmans, L.1
Puntener, D.2
Helenius, A.3
-
77
-
-
0033168382
-
Retrograde protein translocation: ERADication of secretory proteins in health and disease
-
Plemper RK, Wolf DH (1999) Retrograde protein translocation: ERADication of secretory proteins in health and disease. Trends Biochem Sci 24:266-270
-
(1999)
Trends Biochem Sci
, vol.24
, pp. 266-270
-
-
Plemper, R.K.1
Wolf, D.H.2
-
78
-
-
33644847857
-
Clathrin-mediated endocytosis before fluorescent proteins
-
Roth MG (2006) Clathrin-mediated endocytosis before fluorescent proteins. Nat Rev Mol Cell Biol 7:63-68
-
(2006)
Nat Rev Mol Cell Biol
, vol.7
, pp. 63-68
-
-
Roth, M.G.1
-
79
-
-
0034762332
-
Internalization of cholera toxin by different endocytic mechanisms
-
Torgersen ML, Skretting G, Van Deurs B, Sandvig K (2001) Internalization of cholera toxin by different endocytic mechanisms. J Cell Sci 114:3737-3747
-
(2001)
J Cell Sci
, vol.114
, pp. 3737-3747
-
-
Torgersen, M.L.1
Skretting, G.2
Van Deurs, B.3
Sandvig, K.4
-
80
-
-
59249092022
-
Muscular gene transfer using nonviral vectors
-
Braun S (2008) Muscular gene transfer using nonviral vectors. Curr Gene Ther 8:391-405
-
(2008)
Curr Gene Ther
, vol.8
, pp. 391-405
-
-
Braun, S.1
-
81
-
-
0037348748
-
Progress and prospects: Naked DNA gene transfer and therapy
-
Herweijer H, Wolff JA (2003) Progress and prospects: naked DNA gene transfer and therapy. Gene Ther 10:453-458
-
(2003)
Gene Ther
, vol.10
, pp. 453-458
-
-
Herweijer, H.1
Wolff, J.A.2
-
82
-
-
44249123015
-
Ultrasound mediated delivery of drugs and genes to solid tumors
-
Frenkel V (2008) Ultrasound mediated delivery of drugs and genes to solid tumors. Adv Drug Deliv Rev 60:1193-1208
-
(2008)
Adv Drug Deliv Rev
, vol.60
, pp. 1193-1208
-
-
Frenkel, V.1
-
83
-
-
44149099480
-
Ultrasound for drug and gene delivery to the brain
-
Hynynen K (2008) Ultrasound for drug and gene delivery to the brain. Adv Drug Deliv Rev 60:1209-1217
-
(2008)
Adv Drug Deliv Rev
, vol.60
, pp. 1209-1217
-
-
Hynynen, K.1
-
84
-
-
4644371368
-
Gene therapy progress and prospects: Electroporation and other physical methods
-
Wells DJ (2004) Gene therapy progress and prospects: electroporation and other physical methods. Gene Ther 11:1363-1369
-
(2004)
Gene Ther
, vol.11
, pp. 1363-1369
-
-
Wells, D.J.1
-
85
-
-
5444270495
-
DNA electrotransfer: Its principles and an updated review of its therapeutic applications
-
Andre F, Mir LM (2004) DNA electrotransfer: its principles and an updated review of its therapeutic applications. Gene Ther 11 [Suppl 1]: S33-42
-
(2004)
Gene Ther
, vol.11
, pp. S33-S42
-
-
Andre, F.1
Mir, L.M.2
-
87
-
-
0041324854
-
Plasmid-based gene delivery to target tissues in vivo: The intravascular approach
-
Hagstrom JE (2003) Plasmid-based gene delivery to target tissues in vivo: the intravascular approach. Curr Opin Mol Ther 5:338-344
-
(2003)
Curr Opin Mol Ther
, vol.5
, pp. 338-344
-
-
Hagstrom, J.E.1
-
88
-
-
33750626735
-
In vivo electroporation for gene therapy
-
Heller LC, Heller R (2006) In vivo electroporation for gene therapy. Hum Gene Ther 17:890-897
-
(2006)
Hum Gene Ther
, vol.17
, pp. 890-897
-
-
Heller, L.C.1
Heller, R.2
-
89
-
-
34248637892
-
Systemic siRNA delivery via hydrodynamic intravascular injection
-
Lewis DL, Wolff JA (2007) Systemic siRNA delivery via hydrodynamic intravascular injection. Adv Drug Deliv Rev 59:115-123
-
(2007)
Adv Drug Deliv Rev
, vol.59
, pp. 115-123
-
-
Lewis, D.L.1
Wolff, J.A.2
-
90
-
-
0036207351
-
Hyaluronidase increases electrogene transfer efficiency in skeletal muscle
-
Mennuni C, Calvaruso F, Zampaglione I et al (2002) Hyaluronidase increases electrogene transfer efficiency in skeletal muscle. Hum Gene Ther 13:355-365
-
(2002)
Hum Gene Ther
, vol.13
, pp. 355-365
-
-
Mennuni, C.1
Calvaruso, F.2
Zampaglione, I.3
-
91
-
-
44649113067
-
Application of electroporation gene therapy: Past, current, and future
-
Mir LM (2008) Application of electroporation gene therapy: past, current, and future. Methods Mol Biol 423:3-17
-
(2008)
Methods Mol Biol
, vol.423
, pp. 3-17
-
-
Mir, L.M.1
-
92
-
-
33847768717
-
Gene therapy progress and prospects: Ultrasound for gene transfer
-
Newman CM, Bettinger T (2007) Gene therapy progress and prospects: ultrasound for gene transfer. Gene Ther 14:465-475
-
(2007)
Gene Ther
, vol.14
, pp. 465-475
-
-
Newman, C.M.1
Bettinger, T.2
-
93
-
-
70349971779
-
Electroporation advances in large animals
-
in press
-
Reed SD, Li S (2009) Electroporation advances in large animals. Curr Gene Ther (in press)
-
(2009)
Curr Gene Ther
-
-
Reed, S.D.1
Li, S.2
-
94
-
-
37549014128
-
Complete in vivo reversal of the multidrug resistance phenotype by jet-injection of anti-MDR1 short hairpin RNA-encoding plasmid DNA
-
Stein U, Walther W, Stege A et al (2008) Complete in vivo reversal of the multidrug resistance phenotype by jet-injection of anti-MDR1 short hairpin RNA-encoding plasmid DNA. Mol Ther 16:178-186
-
(2008)
Mol Ther
, vol.16
, pp. 178-186
-
-
Stein, U.1
Walther, W.2
Stege, A.3
-
95
-
-
58149337433
-
Novel jet-injection technology for nonviral intratumoral gene transfer in patients with melanoma and breast cancer
-
Walther W, Siegel R, Kobelt D et al (2008) Novel jet-injection technology for nonviral intratumoral gene transfer in patients with melanoma and breast cancer. Clin Cancer Res 14:7545-7553
-
(2008)
Clin Cancer Res
, vol.14
, pp. 7545-7553
-
-
Walther, W.1
Siegel, R.2
Kobelt, D.3
-
96
-
-
0035117313
-
Nonviral in vivo gene delivery into tumors using a novel low volume jet-injection technology
-
Walther W, Stein U, Fichtner I et al (2001) Nonviral in vivo gene delivery into tumors using a novel low volume jet-injection technology. Gene Ther 8:173-180
-
(2001)
Gene Ther
, vol.8
, pp. 173-180
-
-
Walther, W.1
Stein, U.2
Fichtner, I.3
-
97
-
-
0036614970
-
Intratumoral low-volume jet-injection for efficient nonviral gene transfer
-
Walther W, Stein U, Fichtner I et al (2002) Intratumoral low-volume jet-injection for efficient nonviral gene transfer. Mol Biotechnol 21:105-115
-
(2002)
Mol Biotechnol
, vol.21
, pp. 105-115
-
-
Walther, W.1
Stein, U.2
Fichtner, I.3
-
99
-
-
14044250982
-
Formation and intracellular trafficking of lipoplexes and polyplexes
-
Elouahabi A, Ruysschaert JM (2005) Formation and intracellular trafficking of lipoplexes and polyplexes. Mol Ther 11:336-347
-
(2005)
Mol Ther
, vol.11
, pp. 336-347
-
-
Elouahabi, A.1
Ruysschaert, J.M.2
-
100
-
-
13844255516
-
Transcellular protein transduction using the Tat protein of HIV-1
-
Fittipaldi A, Giacca M (2005) Transcellular protein transduction using the Tat protein of HIV-1. Adv Drug Deliv Rev 57:597-608
-
(2005)
Adv Drug Deliv Rev
, vol.57
, pp. 597-608
-
-
Fittipaldi, A.1
Giacca, M.2
-
101
-
-
7444268561
-
The HIV-1 Tat protein: A multifaceted target for novel therapeutic opportunities
-
Giacca M (2004) The HIV-1 Tat protein: a multifaceted target for novel therapeutic opportunities. Curr Drug Targets Immune Endocr Metabol Disord 4:277-285
-
(2004)
Curr Drug Targets Immune Endocr Metabol Disord
, vol.4
, pp. 277-285
-
-
Giacca, M.1
-
102
-
-
33745460260
-
Current status of polymeric gene delivery systems
-
Park TG, Jeong JH, Kim SW (2006) Current status of polymeric gene delivery systems. Adv Drug Deliv Rev 58:467-486
-
(2006)
Adv Drug Deliv Rev
, vol.58
, pp. 467-486
-
-
Park, T.G.1
Jeong, J.H.2
Kim, S.W.3
-
103
-
-
0036731059
-
Influence of the bystander effect on HSV-tk/GCV gene therapy. A review
-
Van Dillen IJ, Mulder NH, Vaalburg W et al (2002) Influence of the bystander effect on HSV-tk/GCV gene therapy. A review. Curr Gene Ther 2:307-322
-
(2002)
Curr Gene Ther
, vol.2
, pp. 307-322
-
-
Van Dillen, I.J.1
Mulder, N.H.2
Vaalburg, W.3
-
104
-
-
0033959447
-
Cancer gene therapy: Hard lessons and new courses
-
Vile RG, Russell SJ, Lemoine NR (2000) Cancer gene therapy: hard lessons and new courses. Gene Ther 7:2-8
-
(2000)
Gene Ther
, vol.7
, pp. 2-8
-
-
Vile, R.G.1
Russell, S.J.2
Lemoine, N.R.3
-
105
-
-
33845190449
-
Cationic lipids, lipoplexes and intracellular delivery of genes
-
Wasungu L, Hoekstra D (2006) Cationic lipids, lipoplexes and intracellular delivery of genes. J Control Release 116:255-264
-
(2006)
J Control Release
, vol.116
, pp. 255-264
-
-
Wasungu, L.1
Hoekstra, D.2
-
106
-
-
4744350485
-
Lipoplex-mediated delivery of nucleic acids: Factors affecting in vivo transfection
-
Dass CR (2004) Lipoplex-mediated delivery of nucleic acids: factors affecting in vivo transfection. J Mol Med 82:579-591
-
(2004)
J Mol Med
, vol.82
, pp. 579-591
-
-
Dass, C.R.1
-
107
-
-
27544477492
-
Intelligent polymers as nonviral vectors
-
Dincer S, Turk M, Piskin E (2005) Intelligent polymers as nonviral vectors. Gene Ther 12 [Suppl 1]: S139-145
-
(2005)
Gene Ther
, vol.12
, pp. S139-S145
-
-
Dincer, S.1
Turk, M.2
Piskin, E.3
-
109
-
-
28744445460
-
Dendrimer biocompatibility and toxicity
-
Duncan R, Izzo L (2005) Dendrimer biocompatibility and toxicity. Adv Drug Deliv Rev 57:2215-2237
-
(2005)
Adv Drug Deliv Rev
, vol.57
, pp. 2215-2237
-
-
Duncan, R.1
Izzo, L.2
-
110
-
-
13844255516
-
Transcellular protein transduction using the Tat protein of HIV-1
-
Fittipaldi A, Giacca M (2005) Transcellular protein transduction using the Tat protein of HIV-1. Adv Drug Deliv Rev 57:597-608
-
(2005)
Adv Drug Deliv Rev
, vol.57
, pp. 597-608
-
-
Fittipaldi, A.1
Giacca, M.2
-
111
-
-
33847014531
-
Gene delivery by cationic lipids: In and out of an endosome
-
Hoekstra D, Rejman J, Wasungu L et al (2007) Gene delivery by cationic lipids: in and out of an endosome. Biochem Soc Trans 35:68-71
-
(2007)
Biochem Soc Trans
, vol.35
, pp. 68-71
-
-
Hoekstra, D.1
Rejman, J.2
Wasungu, L.3
-
112
-
-
34247135913
-
Classical nuclear localization signals: Definition, function, and interaction with importin alpha
-
Lange, A, Mills RE, Lange CJ et al (2007) Classical nuclear localization signals: definition, function, and interaction with importin alpha. J Biol Chem 282:5101-5105
-
(2007)
J Biol Chem
, vol.282
, pp. 5101-5105
-
-
Lange, A.1
Mills, R.E.2
Lange, C.J.3
-
113
-
-
0035946624
-
Cationic lipid-DNA complexes in gene delivery: From biophysics to biological applications
-
Pedroso De Lima MC, Simoes S, Pires P et al (2001) Cationic lipid-DNA complexes in gene delivery: from biophysics to biological applications. Adv Drug Deliv Rev 47:277-294
-
(2001)
Adv Drug Deliv Rev
, vol.47
, pp. 277-294
-
-
Pedroso De Lima, M.C.1
Simoes, S.2
Pires, P.3
-
114
-
-
0034694011
-
A phase III clinical evaluation of herpes simplex virus type 1 thymidine kinase and ganciclovir gene therapy as an adjuvant to surgical resection and radiation in adults with previously untreated glioblastoma multiforme
-
Rainov NG (2000) A phase III clinical evaluation of herpes simplex virus type 1 thymidine kinase and ganciclovir gene therapy as an adjuvant to surgical resection and radiation in adults with previously untreated glioblastoma multiforme. Hum Gene Ther 11:2389-2401
-
(2000)
Hum Gene Ther
, vol.11
, pp. 2389-2401
-
-
Rainov, N.G.1
-
115
-
-
0031463632
-
Therapy of malignant brain tumors by intratumoral implantation of retroviral vector-producing cells
-
Ram Z, Culver KW, Oshiro EM et al (1997) Therapy of malignant brain tumors by intratumoral implantation of retroviral vector-producing cells. Nat Med 3:1354-1361
-
(1997)
Nat Med
, vol.3
, pp. 1354-1361
-
-
Ram, Z.1
Culver, K.W.2
Oshiro, E.M.3
-
116
-
-
60749099362
-
Dendrimers as versatile platform in drug delivery applications
-
Svenson S (2009) Dendrimers as versatile platform in drug delivery applications. Eur J Pharm Biopharm 71:445-462
-
(2009)
Eur J Pharm Biopharm
, vol.71
, pp. 445-462
-
-
Svenson, S.1
-
117
-
-
0034136802
-
Phase I study of adenoviral delivery of the HSV-tk gene and ganciclovir administration in patients with current malignant brain tumors
-
Trask TW, Trask RP, Aguilar-Cordova E et al (2000) Phase I study of adenoviral delivery of the HSV-tk gene and ganciclovir administration in patients with current malignant brain tumors. Mol Ther 1:195-203
-
(2000)
Mol Ther
, vol.1
, pp. 195-203
-
-
Trask, T.W.1
Trask, R.P.2
Aguilar-Cordova, E.3
-
118
-
-
37549032712
-
Breaking the bonds: Non-viral vectors become chemically dynamic
-
Wolff JA, Rozema DB (2008) Breaking the bonds: non-viral vectors become chemically dynamic. Mol Ther 16:8-15
-
(2008)
Mol Ther
, vol.16
, pp. 8-15
-
-
Wolff, J.A.1
Rozema, D.B.2
-
119
-
-
34247181222
-
Gene delivery by cationic lipid vectors: Overcoming cellular barriers
-
Zuhorn IS, Engberts JB, Hoekstra D (2007) Gene delivery by cationic lipid vectors: overcoming cellular barriers. Eur Biophys J 36:349-362
-
(2007)
Eur Biophys J
, vol.36
, pp. 349-362
-
-
Zuhorn, I.S.1
Engberts, J.B.2
Hoekstra, D.3
-
120
-
-
5444275097
-
Immune responses to gene therapy vectors: Influence on vector function and effector mechanisms
-
Bessis N, Garcia Cozar FJ, Boissier MC (2004) Immune responses to gene therapy vectors: influence on vector function and effector mechanisms. Gene Ther 11 [Suppl 1]: S10-17
-
(2004)
Gene Ther
, vol.11
, pp. S10-S17
-
-
Bessis, N.1
Garcia Cozar, F.J.2
Boissier, M.C.3
-
121
-
-
0033999084
-
Gene silencing as a threat to the success of gene therapy
-
Bestor TH (2000) Gene silencing as a threat to the success of gene therapy. J Clin Invest 105:409-411
-
(2000)
J Clin Invest
, vol.105
, pp. 409-411
-
-
Bestor, T.H.1
-
122
-
-
33847205752
-
The genetic engineering of hematopoietic stem cells: The rise of lentiviral vectors, the conundrum of the ltr, and the promise of lineage-restricted vectors
-
Chang AH, Sadelain M (2007) The genetic engineering of hematopoietic stem cells: the rise of lentiviral vectors, the conundrum of the ltr, and the promise of lineage-restricted vectors. Mol Ther 15:445-456
-
(2007)
Mol Ther
, vol.15
, pp. 445-456
-
-
Chang, A.H.1
Sadelain, M.2
-
123
-
-
0004131381
-
-
Cold Spring Harbor Laboratory Press, Cold Spring Harbor, New York, NY, USA
-
Coffin JM, Hughes H, Varmus HE (1997) Retroviruses. Cold Spring Harbor Laboratory Press, Cold Spring Harbor, New York, NY, USA
-
(1997)
Retroviruses
-
-
Coffin, J.M.1
Hughes, H.2
Varmus, H.E.3
-
124
-
-
46249100071
-
Integration site selection by retroviral vectors: Molecular mechanism and clinical consequences
-
Daniel R, Smith JA (2008) Integration site selection by retroviral vectors: molecular mechanism and clinical consequences. Hum Gene Ther 19:557-568
-
(2008)
Hum Gene Ther
, vol.19
, pp. 557-568
-
-
Daniel, R.1
Smith, J.A.2
-
125
-
-
0035135747
-
Viral vectors for gene therapy: The art of turning infectious agents into vehicles of therapeutics
-
Kay MA, Glorioso JC, Naldini L (2001) Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat Med 7:33-40
-
(2001)
Nat Med
, vol.7
, pp. 33-40
-
-
Kay, M.A.1
Glorioso, J.C.2
Naldini, L.3
-
126
-
-
0004250845
-
-
Lippincott Williams & Wilkins, Philadelphia, PA, USA
-
Knipe DM, Roizman B, Howley PM et al (2006) Fields' virology, 5th edn. Lippincott Williams & Wilkins, Philadelphia, PA, USA
-
(2006)
Fields' Virology, 5th Edn
-
-
Knipe, D.M.1
Roizman, B.2
Howley, P.M.3
-
127
-
-
60649103432
-
Clinical application of lentiviral vectors: Concepts and practice
-
Schambach A, Baum C (2008) Clinical application of lentiviral vectors: concepts and practice. Curr Gene Ther 8:474-482
-
(2008)
Curr Gene Ther
, vol.8
, pp. 474-482
-
-
Schambach, A.1
Baum, C.2
-
128
-
-
0041808932
-
Gene therapy progress and prospects: Adenoviral vectors
-
St George JA (2003) Gene therapy progress and prospects: adenoviral vectors. Gene Ther 10:1135-1141
-
(2003)
Gene Ther
, vol.10
, pp. 1135-1141
-
-
St George, J.A.1
-
129
-
-
0038745599
-
Progress and problems with the use of viral vectors for gene therapy
-
Thomas CE, Ehrhardt A, Kay MA (2003) Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet 4:346-358
-
(2003)
Nat Rev Genet
, vol.4
, pp. 346-358
-
-
Thomas, C.E.1
Ehrhardt, A.2
Kay, M.A.3
-
130
-
-
33746874152
-
Adeno-associated virus serotypes: Vector toolkit for human gene therapy
-
Wu Z, Asokan A, Samulski RJ (2006) Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther 14:316-327
-
(2006)
Mol Ther
, vol.14
, pp. 316-327
-
-
Wu, Z.1
Asokan, A.2
Samulski, R.J.3
-
131
-
-
48549098426
-
Adeno-associated virus vectors: Versatile tools for in vivo gene transfer
-
Zentilin L, Giacca M (2008) Adeno-associated virus vectors: versatile tools for in vivo gene transfer. Contrib Nephrol 159:63-77
-
(2008)
Contrib Nephrol
, vol.159
, pp. 63-77
-
-
Zentilin, L.1
Giacca, M.2
-
132
-
-
27944433469
-
Oncolytic viral therapies: The clinical experience
-
Aghi M, Martuza RL (2005) Oncolytic viral therapies: the clinical experience. Oncogene 24:7802-7816
-
(2005)
Oncogene
, vol.24
, pp. 7802-7816
-
-
Aghi, M.1
Martuza, R.L.2
-
133
-
-
0030835321
-
Pseudotyping human immunodeficiency virus type 1 (HIV-1) by the glycoprotein of vesicular stomatitis virus targets HIV-1 entry to an endocytic pathway and suppresses both the requirement for Nef and the sensitivity to cyclosporin A
-
Aiken C (1997) Pseudotyping human immunodeficiency virus type 1 (HIV-1) by the glycoprotein of vesicular stomatitis virus targets HIV-1 entry to an endocytic pathway and suppresses both the requirement for Nef and the sensitivity to cyclosporin A. J Virol 71:5871-5877
-
(1997)
J Virol
, vol.71
, pp. 5871-5877
-
-
Aiken, C.1
-
134
-
-
27544513676
-
Gutless adenovirus: Last-generation adenovirus for gene therapy
-
Alba R, Bosch A, Chillon M (2005) Gutless adenovirus: last-generation adenovirus for gene therapy. Gene Ther 12 [Suppl 1]: S18-27
-
(2005)
Gene Ther
, vol.12
, pp. S18-S27
-
-
Alba, R.1
Bosch, A.2
Chillon, M.3
-
135
-
-
27544463977
-
Replication-competent herpes simplex vectors: Design and applications
-
Argnani R, Lufino M, Manservigi M, Manservigi R (2005) Replication-competent herpes simplex vectors: design and applications. Gene Ther 12 [Suppl 1]: S170-177
-
(2005)
Gene Ther
, vol.12
, pp. S170-S177
-
-
Argnani, R.1
Lufino, M.2
Manservigi, M.3
Manservigi, R.4
-
136
-
-
29144449872
-
Avian sarcoma and leukosis virus-receptor interactions: From classical genetics to novel insights into virus-cell membrane fusion
-
Barnard RJ, Elleder D, Young JA (2006) Avian sarcoma and leukosis virus-receptor interactions: from classical genetics to novel insights into virus-cell membrane fusion. Virology 344:25-29
-
(2006)
Virology
, vol.344
, pp. 25-29
-
-
Barnard, R.J.1
Elleder, D.2
Young, J.A.3
-
137
-
-
5444250395
-
Retroviral vectors: New applications for an old tool
-
Barquinero J, Eixarch H, Perez-Melgosa M (2004) Retroviral vectors: new applications for an old tool. Gene Ther 11 [Suppl 1]: S3-9
-
(2004)
Gene Ther
, vol.11
, pp. S3-S9
-
-
Barquinero, J.1
Eixarch, H.2
Perez-Melgosa, M.3
-
138
-
-
33846015516
-
Transduction of brain by herpes simplex virus vectors
-
Berges BK, Wolfe JH, Fraser NW (2007) Transduction of brain by herpes simplex virus vectors. Mol Ther 15:20-29
-
(2007)
Mol Ther
, vol.15
, pp. 20-29
-
-
Berges, B.K.1
Wolfe, J.H.2
Fraser, N.W.3
-
139
-
-
0029257496
-
The cryptic life style of adeno-associated virus
-
Berns KI, Linden RM (1995) The cryptic life style of adeno-associated virus. BioEssays 17:237-245
-
(1995)
BioEssays
, vol.17
, pp. 237-245
-
-
Berns, K.I.1
Linden, R.M.2
-
140
-
-
27544462820
-
Development and application of replication-incompetent HSV-1-based vectors
-
Berto E, Bozac A, Marconi P (2005) Development and application of replication-incompetent HSV-1-based vectors. Gene Ther 12 [Suppl 1]: S98-102
-
(2005)
Gene Ther
, vol.12
, pp. S98-S102
-
-
Berto, E.1
Bozac, A.2
Marconi, P.3
-
141
-
-
43049178300
-
Progress and prospects: Gene therapy for genetic diseases with helper-dependent adenoviral vectors
-
Brunetti-Pierri N, Ng P (2008) Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors. Gene Ther 15:553-560
-
(2008)
Gene Ther
, vol.15
, pp. 553-560
-
-
Brunetti-Pierri, N.1
Ng, P.2
-
142
-
-
0041808929
-
Receptor targeting of adeno-associated virus vectors
-
Buning H, Ried MU, Perabo L et al (2003) Receptor targeting of adeno-associated virus vectors. Gene Ther 10:1142-1151
-
(2003)
Gene Ther
, vol.10
, pp. 1142-1151
-
-
Buning, H.1
Ried, M.U.2
Perabo, L.3
-
143
-
-
0036812086
-
Replication-defective genomic herpes simplex vectors: Design and production
-
Burton EA, Bai Q, Goins WF, Glorioso JC (2002) Replication-defective genomic herpes simplex vectors: design and production. Curr Opin Biotechnol 13:424-428
-
(2002)
Curr Opin Biotechnol
, vol.13
, pp. 424-428
-
-
Burton, E.A.1
Bai, Q.2
Goins, W.F.3
Glorioso, J.C.4
-
144
-
-
2342660750
-
Integration site selection by retroviruses
-
Cereseto A, Giacca M (2004) Integration site selection by retroviruses. AIDS Rev 6:13-20
-
(2004)
AIDS Rev
, vol.6
, pp. 13-20
-
-
Cereseto, A.1
Giacca, M.2
-
145
-
-
40549138765
-
Processing of recombinant AAV genomes occurs in specific nuclear structures that overlap with foci of DNA-damage-response proteins
-
Cervelli T, Palacios JA, Zentilin L et al (2008) Processing of recombinant AAV genomes occurs in specific nuclear structures that overlap with foci of DNA-damage-response proteins. J Cell Sci 121:349-357
-
(2008)
J Cell Sci
, vol.121
, pp. 349-357
-
-
Cervelli, T.1
Palacios, J.A.2
Zentilin, L.3
-
146
-
-
0032853699
-
Immune responses to adenovirus and adeno-associated virus in humans
-
Chirmule N, Propert K, Magosin S et al (1999) Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther 6:1574-1583
-
(1999)
Gene Ther
, vol.6
, pp. 1574-1583
-
-
Chirmule, N.1
Propert, K.2
Magosin, S.3
-
147
-
-
0033759405
-
Production of first generation adenovirus vectors: A review
-
Danthinne X, Imperiale MJ (2000) Production of first generation adenovirus vectors: a review. Gene Ther 7:1707-1714
-
(2000)
Gene Ther
, vol.7
, pp. 1707-1714
-
-
Danthinne, X.1
Imperiale, M.J.2
-
148
-
-
0031710033
-
A third-generation lentivirus vector with a conditional packaging system
-
Dull T, Zufferey R, Kelly M et al (1998) A third-generation lentivirus vector with a conditional packaging system. J Virol 72:8463-8471
-
(1998)
J Virol
, vol.72
, pp. 8463-8471
-
-
Dull, T.1
Zufferey, R.2
Kelly, M.3
-
149
-
-
0034712849
-
Adeno-associated virus site-specifically integrates into a muscle-specific DNA region
-
Dutheil N, Shi F, Dupressoir T, Linden RM (2000) Adeno-associated virus site-specifically integrates into a muscle-specific DNA region. Proc Natl Acad Sci U S A 97:4862-4866
-
(2000)
Proc Natl Acad Sci U S A
, vol.97
, pp. 4862-4866
-
-
Dutheil, N.1
Shi, F.2
Dupressoir, T.3
Linden, R.M.4
-
150
-
-
27544490857
-
HSV-1-based amplicon vectors: Design and applications
-
Epstein AL (2005) HSV-1-based amplicon vectors: design and applications. Gene Ther 12 [Suppl 1]: S154-158
-
(2005)
Gene Ther
, vol.12
, pp. S154-S158
-
-
Epstein, A.L.1
-
151
-
-
2642539509
-
Gene therapy progress and prospects: Recombinant adeno-associated virus (rAAV) vectors
-
Flotte TR (2004) Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors. Gene Ther 11:805-810
-
(2004)
Gene Ther
, vol.11
, pp. 805-810
-
-
Flotte, T.R.1
-
152
-
-
18444380309
-
Adeno-associated virus: A ubiquitous commensal of mammals
-
Flotte TR, Berns KI (2005) Adeno-associated virus: a ubiquitous commensal of mammals. Hum Gene Ther 16:401-407
-
(2005)
Hum Gene Ther
, vol.16
, pp. 401-407
-
-
Flotte, T.R.1
Berns, K.I.2
-
154
-
-
4043092073
-
Systemic delivery of genes to striated muscles using adeno-associated viral vectors
-
Gregorevic P, Blankinship MJ, Allen JM et al (2004) Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med 10:828-834
-
(2004)
Nat Med
, vol.10
, pp. 828-834
-
-
Gregorevic, P.1
Blankinship, M.J.2
Allen, J.M.3
-
155
-
-
0032506752
-
Novel tools for production and purification of recombinant adenoassociated virus vectors Hum
-
Grimm D, Kern A, Rittner K, Kleinschmidt JA (1998) Novel tools for production and purification of recombinant adenoassociated virus vectors Hum. Gene Ther 9:2745-2760
-
(1998)
Gene Ther
, vol.9
, pp. 2745-2760
-
-
Grimm, D.1
Kern, A.2
Rittner, K.3
Kleinschmidt, J.A.4
-
156
-
-
20844457204
-
Gene targeting with viral vectors
-
Hendrie PC, Russell DW (2005) Gene targeting with viral vectors. Mol Ther 12:9-17
-
(2005)
Mol Ther
, vol.12
, pp. 9-17
-
-
Hendrie, P.C.1
Russell, D.W.2
-
157
-
-
0038805302
-
Immunity to adenovirus and adeno-associated viral vectors: Implications for gene therapy
-
Jooss K, Chirmule N (2003) Immunity to adenovirus and adeno-associated viral vectors: implications for gene therapy. Gene Ther 10:955-963
-
(2003)
Gene Ther
, vol.10
, pp. 955-963
-
-
Jooss, K.1
Chirmule, N.2
-
158
-
-
0029858907
-
Site-specific integration by adeno-associated virus
-
Linden RM, Ward P, Giraud C et al (1996) Site-specific integration by adeno-associated virus. Proc Natl Acad Sci U S A 93:11288-11294
-
(1996)
Proc Natl Acad Sci U S A
, vol.93
, pp. 11288-11294
-
-
Linden, R.M.1
Ward, P.2
Giraud, C.3
-
159
-
-
0037790710
-
Molecular basis of the inflammatory response to adenovirus vectors
-
Liu Q, Muruve DA (2003) Molecular basis of the inflammatory response to adenovirus vectors. Gene Ther 10:935-940
-
(2003)
Gene Ther
, vol.10
, pp. 935-940
-
-
Liu, Q.1
Muruve, D.A.2
-
160
-
-
84920082511
-
Concerted action of cellular JNK and Pin1 restricts HIV-1 genome integration to activated CD4+ T lymphocytes
-
Manganaro L, Lusic M, Gutierrez MI et al (2009) Concerted action of cellular JNK and Pin1 restricts HIV-1 genome integration to activated CD4+ T lymphocytes. Nat Med
-
(2009)
Nat Med
-
-
Manganaro, L.1
Lusic, M.2
Gutierrez, M.I.3
-
161
-
-
45849112549
-
HSV as a vector in vaccine development and gene therapy
-
Marconi P, Argnani R, Berto E et al (2008) HSV as a vector in vaccine development and gene therapy. Hum Vaccin 4:91-105
-
(2008)
Hum Vaccin
, vol.4
, pp. 91-105
-
-
Marconi, P.1
Argnani, R.2
Berto, E.3
-
162
-
-
10944269753
-
Integration of adeno-associated virus (AAV) and recombinant AAV vectors
-
McCarty DM, Young SM Jr, Samulski RJ (2004) Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Annu Rev Genet 38:819-845
-
(2004)
Annu Rev Genet
, vol.38
, pp. 819-845
-
-
McCarty, D.M.1
Young, S.M.2
Samulski, R.J.3
-
163
-
-
27544445072
-
Current issues in adeno-associated viral vector production
-
Merten OW, Geny-Fiamma C, Douar AM (2005) Current issues in adeno-associated viral vector production. Gene Ther 12 [Suppl 1]: S51-61
-
(2005)
Gene Ther
, vol.12
, pp. S51-S61
-
-
Merten, O.W.1
Geny-Fiamma, C.2
Douar, A.M.3
-
164
-
-
0025398116
-
Retrovirus packaging cells
-
Miller AD (1990) Retrovirus packaging cells. Hum Gene Ther 1:5-14
-
(1990)
Hum Gene Ther
, vol.1
, pp. 5-14
-
-
Miller, A.D.1
-
165
-
-
0029907958
-
Cell-surface receptors for retroviruses and implications for gene transfer
-
Miller AD (1996) Cell-surface receptors for retroviruses and implications for gene transfer. Proc Natl Acad Sci U S A 93:11407-11413
-
(1996)
Proc Natl Acad Sci U S A
, vol.93
, pp. 11407-11413
-
-
Miller, A.D.1
-
166
-
-
33747046320
-
Gene targeting in vivo by adeno-associated virus vectors
-
Miller DG, Wang PR, Petek LM et al (2006) Gene targeting in vivo by adeno-associated virus vectors. Nature Biotech 24:1022-1026
-
(2006)
Nature Biotech
, vol.24
, pp. 1022-1026
-
-
Miller, D.G.1
Wang, P.R.2
Petek, L.M.3
-
167
-
-
44049094771
-
Clinical gene therapy using recombinant adeno-associated virus vectors
-
Mueller C, Flotte TR (2008) Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther 15:858-863
-
(2008)
Gene Ther
, vol.15
, pp. 858-863
-
-
Mueller, C.1
Flotte, T.R.2
-
168
-
-
0029996147
-
In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
-
Naldini L, Blomer U, Gallay P et al (1996) In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272:263-267
-
(1996)
Science
, vol.272
, pp. 263-267
-
-
Naldini, L.1
Blomer, U.2
Gallay, P.3
-
169
-
-
33646887376
-
Genotoxicity of retroviral integration in hematopoietic cells
-
Nienhuis AW, Dunbar CE, Sorrentino BP (2006) Genotoxicity of retroviral integration in hematopoietic cells. Mol Ther 13:1031-1049
-
(2006)
Mol Ther
, vol.13
, pp. 1031-1049
-
-
Nienhuis, A.W.1
Dunbar, C.E.2
Sorrentino, B.P.3
-
170
-
-
0031897270
-
Human gene targeting by viral vectors
-
Russell DW, Hirata RK (1998) Human gene targeting by viral vectors. Nat Genet 18:325-330
-
(1998)
Nat Genet
, vol.18
, pp. 325-330
-
-
Russell, D.W.1
Hirata, R.K.2
-
171
-
-
0033756217
-
Update on adenovirus and its vectors
-
Russell WC (2000) Update on adenovirus and its vectors. J Gen Virol 81:2573-2604
-
(2000)
J Gen Virol
, vol.81
, pp. 2573-2604
-
-
Russell, W.C.1
-
172
-
-
59849102099
-
Adenoviruses: Update on structure and function
-
Russell WC (2009) Adenoviruses: update on structure and function. J Gen Virol 90:1-20
-
(2009)
J Gen Virol
, vol.90
, pp. 1-20
-
-
Russell, W.C.1
-
173
-
-
0010390169
-
Cloning of adeno-associated virus into pBR322: Rescue of intact virus from the recombinant plasmid in human cells
-
Samulski RJ, Berns KI, Tan M, Muzyczka N (1982) Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells. Proc Natl Acad Sci USA 79:2077-2081
-
(1982)
Proc Natl Acad Sci USA
, vol.79
, pp. 2077-2081
-
-
Samulski, R.J.1
Berns, K.I.2
Tan, M.3
Muzyczka, N.4
-
174
-
-
0037162715
-
HIV-1 integration in the human genome favors active genes and local hotspots
-
Schroder AR, Shinn P, Chen H et al (2002) HIV-1 integration in the human genome favors active genes and local hotspots. Cell 110:521-529
-
(2002)
Cell
, vol.110
, pp. 521-529
-
-
Schroder, A.R.1
Shinn, P.2
Chen, H.3
-
175
-
-
22744455256
-
Gene therapy progress and prospects: Development of improved lentiviral and retroviral vectors: Design, biosafety, and production
-
Sinn PL, Sauter SL, McCray PB Jr (2005) Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors: design, biosafety, and production. Gene Ther 12:1089-1098
-
(2005)
Gene Ther
, vol.12
, pp. 1089-1098
-
-
Sinn, P.L.1
Sauter, S.L.2
Mccray, P.B.3
-
176
-
-
28144437766
-
Precise hit: Adeno-associated virus in gene targeting
-
Vasileva A, Jessberger R (2005) Precise hit: adeno-associated virus in gene targeting. Nat Rev Microbiol 3:837-847
-
(2005)
Nat Rev Microbiol
, vol.3
, pp. 837-847
-
-
Vasileva, A.1
Jessberger, R.2
-
177
-
-
2942650156
-
Pathways of cell infection by parvoviruses and adeno-associated viruses
-
Vihinen-Ranta M, Suikkanen S, Parrish CR (2004) Pathways of cell infection by parvoviruses and adeno-associated viruses. J Virol 78:6709-6714
-
(2004)
J Virol
, vol.78
, pp. 6709-6714
-
-
Vihinen-Ranta, M.1
Suikkanen, S.2
Parrish, C.R.3
-
178
-
-
19644378907
-
Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart
-
Wang Z, Zhu T, Qiao C et al (2005) Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat Biotechnol 23:321-328
-
(2005)
Nat Biotechnol
, vol.23
, pp. 321-328
-
-
Wang, Z.1
Zhu, T.2
Qiao, C.3
-
179
-
-
41749106705
-
Prospects for the therapeutic application of lentivirusbased gene therapy to HIV-1 infection
-
Yamamoto T, Tsunetsugu-Yokota Y (2008) Prospects for the therapeutic application of lentivirusbased gene therapy to HIV-1 infection. Curr Gene Ther 8:1-8
-
(2008)
Curr Gene Ther
, vol.8
, pp. 1-8
-
-
Yamamoto, T.1
Tsunetsugu-Yokota, Y.2
-
180
-
-
0033951116
-
Variegation of retroviral vector gene expression in myeloid cells
-
Zentilin L, Qin G, Tafuro S et al (2000) Variegation of retroviral vector gene expression in myeloid cells. Gene Ther 7:153-166
-
(2000)
Gene Ther
, vol.7
, pp. 153-166
-
-
Zentilin, L.1
Qin, G.2
Tafuro, S.3
-
181
-
-
0031743608
-
Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery
-
Zufferey R, Dull T, Mandel RJ et al (1998) Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J Virol 72:9873-9880
-
(1998)
J Virol
, vol.72
, pp. 9873-9880
-
-
Zufferey, R.1
Dull, T.2
Mandel, R.J.3
-
182
-
-
34848902043
-
Progress and prospects: Gene therapy clinical trials (part 1)
-
Alexander BL, Ali RR, Alton EW et al (2007) Progress and prospects: gene therapy clinical trials (part 1). Gene Ther 14:1439-1447
-
(2007)
Gene Ther
, vol.14
, pp. 1439-1447
-
-
Alexander, B.L.1
Ali, R.R.2
Alton, E.W.3
-
183
-
-
35848965496
-
Progress and prospects: Gene therapy clinical trials (part 2)
-
Alton E, Ferrari S, Griesenbach U (2007) Progress and prospects: gene therapy clinical trials (part 2). Gene Ther 14:1555-1563
-
(2007)
Gene Ther
, vol.14
, pp. 1555-1563
-
-
Alton, E.1
Ferrari, S.2
Griesenbach, U.3
-
184
-
-
35148817461
-
Gene therapy clinical trials worldwide to 2007: An update
-
Edelstein ML, Abedi MR, Wixon J (2007) Gene therapy clinical trials worldwide to 2007: an update. J Gene Med 9:833-842
-
(2007)
J Gene Med
, vol.9
, pp. 833-842
-
-
Edelstein, M.L.1
Abedi, M.R.2
Wixon, J.3
-
186
-
-
44849140430
-
Gene therapy of inherited diseases
-
Fischer A, Cavazzana-Calvo M (2008) Gene therapy of inherited diseases. Lancet 371:2044-2047
-
(2008)
Lancet
, vol.371
, pp. 2044-2047
-
-
Fischer, A.1
Cavazzana-Calvo, M.2
-
187
-
-
33749383278
-
A look to future directions in gene therapy research for monogenic diseases
-
Porteus MH, Connelly JP, Pruett SM (2006) A look to future directions in gene therapy research for monogenic diseases. PLoS Genet 2:e133
-
(2006)
PLoS Genet
, vol.2
, pp. e133
-
-
Porteus, M.H.1
Connelly, J.P.2
Pruett, S.M.3
-
188
-
-
4944255621
-
Recent developments and current status of gene therapy using viral vectors in the United Kingdom
-
Relph K, Harrington K, Pandha H (2004) Recent developments and current status of gene therapy using viral vectors in the United Kingdom. BMJ 329:839-842
-
(2004)
BMJ
, vol.329
, pp. 839-842
-
-
Relph, K.1
Harrington, K.2
Pandha, H.3
-
189
-
-
35148855074
-
An inventory of shedding data from clinical gene therapy trials
-
Schenk-Braat EA, Van Mierlo MM, Wagemaker G et al (2007) An inventory of shedding data from clinical gene therapy trials. J Gene Med 9:910-921
-
(2007)
J Gene Med
, vol.9
, pp. 910-921
-
-
Schenk-Braat, E.A.1
Van Mierlo, M.M.2
Wagemaker, G.3
-
191
-
-
35348850734
-
Hematopoietic cytokine receptor signaling
-
Baker SJ, Rane SG, Reddy EP (2007) Hematopoietic cytokine receptor signaling. Oncogene 26:6724-6737
-
(2007)
Oncogene
, vol.26
, pp. 6724-6737
-
-
Baker, S.J.1
Rane, S.G.2
Reddy, E.P.3
-
192
-
-
34249890562
-
Gene therapy for severe combined immunodeficiency: Are we there yet?
-
Cavazzana-Calvo M, Fischer A (2007) Gene therapy for severe combined immunodeficiency: are we there yet? J Clin Invest 117:1456-1465
-
(2007)
J Clin Invest
, vol.117
, pp. 1456-1465
-
-
Cavazzana-Calvo, M.1
Fischer, A.2
-
194
-
-
37749025173
-
Gene therapy approaches for stem cell protection
-
Greenberger JS (2008) Gene therapy approaches for stem cell protection. Gene Ther 15:100-108
-
(2008)
Gene Ther
, vol.15
, pp. 100-108
-
-
Greenberger, J.S.1
-
195
-
-
0035991657
-
Of mice and men: The tale of two therapies
-
Hawley RG, Sobieski DA (2002) Of mice and men: the tale of two therapies. Stem Cells 20:275-278
-
(2002)
Stem Cells
, vol.20
, pp. 275-278
-
-
Hawley, R.G.1
Sobieski, D.A.2
-
196
-
-
0036613395
-
Gene therapy of hematopoietic stem cells: Strategies for improvement
-
Hossle JP, Seger RA, Steinhoff D (2002) Gene therapy of hematopoietic stem cells: strategies for improvement. News Physiol Sci 17:87-92
-
(2002)
News Physiol Sci
, vol.17
, pp. 87-92
-
-
Hossle, J.P.1
Seger, R.A.2
Steinhoff, D.3
-
197
-
-
0030989006
-
In vivo drug-selectable genes: A new concept in gene therapy
-
Licht T, Herrmann F, Gottesman MM, Pastan I (1997) In vivo drug-selectable genes: a new concept in gene therapy. Stem Cells 15:104-111
-
(1997)
Stem Cells
, vol.15
, pp. 104-111
-
-
Licht, T.1
Herrmann, F.2
Gottesman, M.M.3
Pastan, I.4
-
198
-
-
44849127181
-
Development of gene therapy for blood disorders
-
Nienhuis AW (2008) Development of gene therapy for blood disorders. Blood 111:4431-4444
-
(2008)
Blood
, vol.111
, pp. 4431-4444
-
-
Nienhuis, A.W.1
-
199
-
-
33645861655
-
Gene therapy for lysosomal storage diseases
-
Sands MS, Davidson BL (2006) Gene therapy for lysosomal storage diseases. Mol Ther 13:839-849
-
(2006)
Mol Ther
, vol.13
, pp. 839-849
-
-
Sands, M.S.1
Davidson, B.L.2
-
200
-
-
33947210724
-
The continuing contribution of gene marking to cell and gene therapy
-
Tey SK, Brenner MK (2007) The continuing contribution of gene marking to cell and gene therapy. Mol Ther 15:666-676
-
(2007)
Mol Ther
, vol.15
, pp. 666-676
-
-
Tey, S.K.1
Brenner, M.K.2
-
201
-
-
33748939587
-
Gene therapy: X-SCID transgene leukaemogenicity
-
discussion E6-7
-
Thrasher AJ, Gaspar HB, Baum C et al (2006) Gene therapy: X-SCID transgene leukaemogenicity. Nature 443:E5-6; discussion E6-7
-
(2006)
Nature
, vol.443
, pp. E5-E6
-
-
Thrasher, A.J.1
Gaspar, H.B.2
Baum, C.3
-
202
-
-
4344693571
-
Cytokines: Value-added products in hematopoietic stem cell gene therapy
-
Zielske SP, Braun SE (2004) Cytokines: value-added products in hematopoietic stem cell gene therapy. Mol Ther 10:211-219
-
(2004)
Mol Ther
, vol.10
, pp. 211-219
-
-
Zielske, S.P.1
Braun, S.E.2
-
203
-
-
59449098985
-
Gene therapy for immunodeficiency due to adenosine deaminase deficiency
-
Aiuti A, Cattaneo F, Galimberti S et al (2009) Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 360:447-458
-
(2009)
N Engl J Med
, vol.360
, pp. 447-458
-
-
Aiuti, A.1
Cattaneo, F.2
Galimberti, S.3
-
204
-
-
0037189401
-
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
-
Aiuti A, Slavin S, Aker M et al (2002) Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 296:2410-2413
-
(2002)
Science
, vol.296
, pp. 2410-2413
-
-
Aiuti, A.1
Slavin, S.2
Aker, M.3
-
205
-
-
18444376150
-
Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement
-
Aiuti A, Vai S, Mortellaro A et al (2002) Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement. Nat Med 8:423-425
-
(2002)
Nat Med
, vol.8
, pp. 423-425
-
-
Aiuti, A.1
Vai, S.2
Mortellaro, A.3
-
207
-
-
10744233606
-
Chance or necessity? Insertional mutagenesis in gene therapy and its consequences
-
Baum C, Von Kalle C, Staal FJ et al (2004) Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. Mol Ther 9:5-13
-
(2004)
Mol Ther
, vol.9
, pp. 5-13
-
-
Baum, C.1
Von Kalle, C.2
Staal, F.J.3
-
208
-
-
33947198645
-
New therapeutic options for lysosomal storage disorders: Enzyme replacement, small molecules and gene therapy
-
Beck M (2007) New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy. Hum Genet 121:1-22
-
(2007)
Hum Genet
, vol.121
, pp. 1-22
-
-
Beck, M.1
-
209
-
-
26644436945
-
Gene therapy of storage disorders by retroviral and lentiviral vectors
-
Biffi A, Naldini L (2005) Gene therapy of storage disorders by retroviral and lentiviral vectors. Hum Gene Ther 16:1133-1142
-
(2005)
Hum Gene Ther
, vol.16
, pp. 1133-1142
-
-
Biffi, A.1
Naldini, L.2
-
210
-
-
34250718593
-
The suicide gene therapy challenge: How to improve a successful gene therapy approach
-
Bonini C, Bondanza A, Perna SK et al (2007) The suicide gene therapy challenge: how to improve a successful gene therapy approach. Mol Ther 15:1248-1252
-
(2007)
Mol Ther
, vol.15
, pp. 1248-1252
-
-
Bonini, C.1
Bondanza, A.2
Perna, S.K.3
-
211
-
-
0030124147
-
Gene marking
-
Brenner MK (1996) Gene marking. Gene Ther 3:278-279
-
(1996)
Gene Ther
, vol.3
, pp. 278-279
-
-
Brenner, M.K.1
-
212
-
-
34547657665
-
Hot spots of retroviral integration in human CD34+ hematopoietic cells
-
Cattoglio C, Facchini G, Sartori D et al (2007) Hot spots of retroviral integration in human CD34+ hematopoietic cells. Blood 110:1770-1778
-
(2007)
Blood
, vol.110
, pp. 1770-1778
-
-
Cattoglio, C.1
Facchini, G.2
Sartori, D.3
-
213
-
-
0034724857
-
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
-
Cavazzana-Calvo M, Hacein-Bey S, De Saint Basile G et al (2000) Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288:669-672
-
(2000)
Science
, vol.288
, pp. 669-672
-
-
Cavazzana-Calvo, M.1
Hacein-Bey, S.2
De Saint Basile, G.3
-
214
-
-
0042526080
-
Gene therapy progress and prospects: Gene therapy of lysosomal storage disorders
-
Cheng SH, Smith AE (2003) Gene therapy progress and prospects: gene therapy of lysosomal storage disorders. Gene Ther 10:1275-1281
-
(2003)
Gene Ther
, vol.10
, pp. 1275-1281
-
-
Cheng, S.H.1
Smith, A.E.2
-
215
-
-
0347634456
-
Gene therapy insertional mutagenesis insights
-
Dave UP, Jenkins NA, Copeland NG (2004) Gene therapy insertional mutagenesis insights. Science 303:333
-
(2004)
Science
, vol.303
, pp. 333
-
-
Dave, U.P.1
Jenkins, N.A.2
Copeland, N.G.3
-
216
-
-
85041132416
-
Genetic marking shows that Ph+ cells present in autologous transplants of chronic myelogenous leukemia (CML) contribute to relapse after autologous bone marrow in CML
-
Deisseroth AB, Zu Z, Claxton D et al (1994) Genetic marking shows that Ph+ cells present in autologous transplants of chronic myelogenous leukemia (CML) contribute to relapse after autologous bone marrow in CML. Blood 83:3068-3076
-
(1994)
Blood
, vol.83
, pp. 3068-3076
-
-
Deisseroth, A.B.1
Zu, Z.2
Claxton, D.3
-
217
-
-
0026628807
-
Chronic granulomatous disease
-
Dinauer MC, Orkin SH (1992) Chronic granulomatous disease. Annu Rev Med 43:117-124
-
(1992)
Annu Rev Med
, vol.43
, pp. 117-124
-
-
Dinauer, M.C.1
Orkin, S.H.2
-
218
-
-
33748413936
-
Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning
-
Gaspar HB, Bjorkegren E, Parsley K et al (2006) Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. Mol Ther 14:505-513
-
(2006)
Mol Ther
, vol.14
, pp. 505-513
-
-
Gaspar, H.B.1
Bjorkegren, E.2
Parsley, K.3
-
219
-
-
19944421384
-
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector
-
Gaspar HB, Parsley KL, Howe S et al (2004) Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 364:2181-2187
-
(2004)
Lancet
, vol.364
, pp. 2181-2187
-
-
Gaspar, H.B.1
Parsley, K.L.2
Howe, S.3
-
220
-
-
0037448352
-
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
-
Hacein-Bey-Abina S, Von Kalle C, Schmidt M et al (2003) A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 348:255-256
-
(2003)
N Engl J Med
, vol.348
, pp. 255-256
-
-
Hacein-Bey-Abina, S.1
Von Kalle, C.2
Schmidt, M.3
-
221
-
-
0142084745
-
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
-
Hacein-Bey-Abina S, Von Kalle C, Schmidt M et al (2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302:415-419
-
(2003)
Science
, vol.302
, pp. 415-419
-
-
Hacein-Bey-Abina, S.1
Von Kalle, C.2
Schmidt, M.3
-
222
-
-
33646195657
-
Cell and gene-based therapies for the lysosomal storage diseases
-
Hodges BL, Cheng SH (2006) Cell and gene-based therapies for the lysosomal storage diseases. Curr Gene Ther 6:227-241
-
(2006)
Curr Gene Ther
, vol.6
, pp. 227-241
-
-
Hodges, B.L.1
Cheng, S.H.2
-
223
-
-
0042738935
-
Occurrence of leukaemia following gene therapy of X-linked SCID
-
Kohn DB, Sadelain M, Glorioso JC (2003) Occurrence of leukaemia following gene therapy of X-linked SCID. Nat Rev Cancer 3:477-488
-
(2003)
Nat Rev Cancer
, vol.3
, pp. 477-488
-
-
Kohn, D.B.1
Sadelain, M.2
Glorioso, J.C.3
-
224
-
-
0038446699
-
Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: Long-term results of the first clinical gene therapy trial
-
Muul LM, Tuschong LM, Soenen SL et al (2003) Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial. Blood 101:2563-2569
-
(2003)
Blood
, vol.101
, pp. 2563-2569
-
-
Muul, L.M.1
Tuschong, L.M.2
Soenen, S.L.3
-
225
-
-
33645734405
-
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
-
Ott MG, Schmidt M, Schwarzwaelder K et al (2006) Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 12:401-409
-
(2006)
Nat Med
, vol.12
, pp. 401-409
-
-
Ott, M.G.1
Schmidt, M.2
Schwarzwaelder, K.3
-
226
-
-
34547651095
-
Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo
-
Schwarzwaelder K, Howe SJ, Schmidt M et al (2007) Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. J Clin Invest 117:2241-2249
-
(2007)
J Clin Invest
, vol.117
, pp. 2241-2249
-
-
Schwarzwaelder, K.1
Howe, S.J.2
Schmidt, M.3
-
227
-
-
33746801652
-
Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy
-
Shou Y, Ma Z, Lu T, Sorrentino BP (2006) Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy. Proc Natl Acad Sci U S A 103:11730-11735
-
(2006)
Proc Natl Acad Sci U S A
, vol.103
, pp. 11730-11735
-
-
Shou, Y.1
Ma, Z.2
Lu, T.3
Sorrentino, B.P.4
-
228
-
-
33646361846
-
Gene therapy: Therapeutic gene causing lymphoma
-
Woods NB, Bottero V, Schmidt M et al (2006) Gene therapy: therapeutic gene causing lymphoma. Nature 440:1123
-
(2006)
Nature
, vol.440
, pp. 1123
-
-
Woods, N.B.1
Bottero, V.2
Schmidt, M.3
-
229
-
-
33646191190
-
Gene therapy for cystic fibrosis airway disease: Is clinical success imminent?
-
Anson DS, Smith GJ, Parsons DW (2006) Gene therapy for cystic fibrosis airway disease: is clinical success imminent? Curr Gene Ther 6:161-179
-
(2006)
Curr Gene Ther
, vol.6
, pp. 161-179
-
-
Anson, D.S.1
Smith, G.J.2
Parsons, D.W.3
-
230
-
-
33846271820
-
Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis
-
Flotte TR, Ng P, Dylla DE et al (2007) Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis. Mol Ther 15:229-241
-
(2007)
Mol Ther
, vol.15
, pp. 229-241
-
-
Flotte, T.R.1
Ng, P.2
Dylla, D.E.3
-
231
-
-
60149095412
-
Gene transfer to the lung: Lessons learned from more than 2 decades of CF gene therapy
-
Griesenbach U, Alton EW (2009) Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapy. Adv Drug Deliv Rev 61:128-139
-
(2009)
Adv Drug Deliv Rev
, vol.61
, pp. 128-139
-
-
Griesenbach, U.1
Alton, E.W.2
-
232
-
-
33745755929
-
Gene therapy progress and prospects: Cystic fibrosis
-
Griesenbach U, Geddes DM, Alton EW (2006) Gene therapy progress and prospects: cystic fibrosis. Gene Ther 13:1061-1067
-
(2006)
Gene Ther
, vol.13
, pp. 1061-1067
-
-
Griesenbach, U.1
Geddes, D.M.2
Alton, E.W.3
-
234
-
-
50649123290
-
CFTR function and prospects for therapy
-
Riordan JR (2008) CFTR function and prospects for therapy. Annu Rev Biochem 77:701-726
-
(2008)
Annu Rev Biochem
, vol.77
, pp. 701-726
-
-
Riordan, J.R.1
-
235
-
-
0028168641
-
Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis
-
Crystal RG, McElvaney NG, Rosenfeld MA et al (1994) Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nat Genet 8:42-51
-
(1994)
Nat Genet
, vol.8
, pp. 42-51
-
-
Crystal, R.G.1
McElvaney, N.G.2
Rosenfeld, M.A.3
-
237
-
-
34249090543
-
Gene delivery to airway epithelial cells in vivo: A direct comparison of apical and basolateral transduction strategies using pseudotyped lentivirus vectors
-
Kremer KL, Dunning KR, Parsons DW, Anson DS (2007) Gene delivery to airway epithelial cells in vivo: a direct comparison of apical and basolateral transduction strategies using pseudotyped lentivirus vectors. J Gene Med 9:362-368
-
(2007)
J Gene Med
, vol.9
, pp. 362-368
-
-
Kremer, K.L.1
Dunning, K.R.2
Parsons, D.W.3
Anson, D.S.4
-
238
-
-
73849130017
-
Generation of novel AAV variants by directed evolution for improved CFTR delivery to human ciliated airway epithelium
-
Li W, Zhang L, Johnson JS et al (2009) Generation of novel AAV variants by directed evolution for improved CFTR delivery to human ciliated airway epithelium. Mol Ther 17:2067-2077
-
(2009)
Mol Ther
, vol.17
, pp. 2067-2077
-
-
Li, W.1
Zhang, L.2
Johnson, J.S.3
-
239
-
-
42549138758
-
A receptor-targeted nanocomplex vector system optimized for respiratory gene transfer
-
Tagalakis AD, McAnulty RJ, Devaney J et al (2008) A receptor-targeted nanocomplex vector system optimized for respiratory gene transfer. Mol Ther 16:907-915
-
(2008)
Mol Ther
, vol.16
, pp. 907-915
-
-
Tagalakis, A.D.1
McAnulty, R.J.2
Devaney, J.3
-
240
-
-
5444275698
-
Recombinant adeno-associated viral (rAAV) vectors as therapeutic tools for Duchenne muscular dystrophy (DMD)
-
Athanasopoulos T, Graham IR, Foster H, Dickson G (2004) Recombinant adeno-associated viral (rAAV) vectors as therapeutic tools for Duchenne muscular dystrophy (DMD). Gene Ther 11 [Suppl 1]: S109-121
-
(2004)
Gene Ther
, vol.11
, pp. S109-S121
-
-
Athanasopoulos, T.1
Graham, I.R.2
Foster, H.3
Dickson, G.4
-
241
-
-
20444475788
-
Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies
-
Chakkalakal J V, Thompson J, Parks RJ, Jasmin BJ (2005) Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies. FASEB J 19:880-891
-
(2005)
FASEB J
, vol.19
, pp. 880-891
-
-
Chakkalakal, J.V.1
Thompson, J.2
Parks, R.J.3
Jasmin, B.J.4
-
242
-
-
33845218878
-
Gene therapy progress and prospects: Duchenne muscular dystrophy
-
Foster K, Foster H, Dickson JG (2006) Gene therapy progress and prospects: Duchenne muscular dystrophy. Gene Ther 13:1677-1685
-
(2006)
Gene Ther
, vol.13
, pp. 1677-1685
-
-
Foster, K.1
Foster, H.2
Dickson, J.G.3
-
243
-
-
32244443828
-
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
-
Alter J, Lou F, Rabinowitz A et al (2006) Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med 12:175-177
-
(2006)
Nat Med
, vol.12
, pp. 175-177
-
-
Alter, J.1
Lou, F.2
Rabinowitz, A.3
-
244
-
-
10744233235
-
Dystrophic phenotype of canine X-linked muscular dystrophy is mitigated by adenovirus-mediated utrophin gene transfer
-
Cerletti M, Negri T, Cozzi F et al (2003) Dystrophic phenotype of canine X-linked muscular dystrophy is mitigated by adenovirus-mediated utrophin gene transfer. Gene Ther 10:750-757
-
(2003)
Gene Ther
, vol.10
, pp. 750-757
-
-
Cerletti, M.1
Negri, T.2
Cozzi, F.3
-
245
-
-
33749004495
-
Challenges and opportunities in dystrophin-deficient cardiomyopathy gene therapy
-
Duan D (2006) Challenges and opportunities in dystrophin-deficient cardiomyopathy gene therapy. Hum Mol Genet 15 [Spec No 2]: R253-261
-
(2006)
Hum Mol Genet
, vol.15
, Issue.2
, pp. R253-R261
-
-
Duan, D.1
-
246
-
-
10044240371
-
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
-
Goyenvalle A, Vulin A, Fougerousse F et al (2004) Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science 306:1796-1799
-
(2004)
Science
, vol.306
, pp. 1796-1799
-
-
Goyenvalle, A.1
Vulin, A.2
Fougerousse, F.3
-
247
-
-
4043092073
-
Systemic delivery of genes to striated muscles using adeno-associated viral vectors
-
Gregorevic P, Blankinship MJ, Allen JM et al (2004) Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med 10:828-834
-
(2004)
Nat Med
, vol.10
, pp. 828-834
-
-
Gregorevic, P.1
Blankinship, M.J.2
Allen, J.M.3
-
248
-
-
70349451258
-
Development of antisense-mediated exon skipping as a treatment for duchenne muscular dystrophy
-
Heemskerk H, De Winter CL, Van Ommen GJ et al (2009) Development of antisense-mediated exon skipping as a treatment for duchenne muscular dystrophy. Ann N Y Acad Sci 1175:71-79
-
(2009)
Ann N Y Acad Sci
, vol.1175
, pp. 71-79
-
-
Heemskerk, H.1
De Winter, C.L.2
Van Ommen, G.J.3
-
249
-
-
11844256373
-
Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles
-
Lu QL, Rabinowitz A, Chen YC et al (2005) Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci U S A 102:198-203
-
(2005)
Proc Natl Acad Sci U S A
, vol.102
, pp. 198-203
-
-
Lu, Q.L.1
Rabinowitz, A.2
Chen, Y.C.3
-
250
-
-
70350067897
-
Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins
-
Mendell JR, Rodino-Klapac LR, Rosales-Quintero X et al (2009) Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Ann Neurol 66:290-297
-
(2009)
Ann Neurol
, vol.66
, pp. 290-297
-
-
Mendell, J.R.1
Rodino-Klapac, L.R.2
Rosales-Quintero, X.3
-
251
-
-
7544232737
-
Utrophin therapy for Duchenne muscular dystrophy?
-
Nelson R (2004) Utrophin therapy for Duchenne muscular dystrophy? Lancet Neurol 3:637
-
(2004)
Lancet Neurol
, vol.3
, pp. 637
-
-
Nelson, R.1
-
252
-
-
6344263893
-
Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy
-
Romero NB, Braun S, Benveniste O et al (2004) Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy. Hum Gene Ther 15:1065-1076
-
(2004)
Hum Gene Ther
, vol.15
, pp. 1065-1076
-
-
Romero, N.B.1
Braun, S.2
Benveniste, O.3
-
253
-
-
0036823730
-
Viral vectors for gene transfer of micro-, mini-, or fulllength dystrophin
-
Scott JM, Li S, Harper SQ et al (2002) Viral vectors for gene transfer of micro-, mini-, or fulllength dystrophin. Neuromuscul Disord 12 [Suppl 1]: S23-29
-
(2002)
Neuromuscul Disord
, vol.12
, pp. S23-S29
-
-
Scott, J.M.1
Li, S.2
Harper, S.Q.3
-
254
-
-
37549034298
-
Local dystrophin restoration with antisense oligonucleotide PRO051
-
Van Deutekom JC, Janson AA, Ginjaar IB et al (2007) Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med 357:2677-2686
-
(2007)
N Engl J Med
, vol.357
, pp. 2677-2686
-
-
Van Deutekom, J.C.1
Janson, A.A.2
Ginjaar, I.B.3
-
255
-
-
19644378907
-
Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart
-
Wang Z, Zhu T, Qiao C et al (2005) Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat Biotechnol 23:321-328
-
(2005)
Nat Biotechnol
, vol.23
, pp. 321-328
-
-
Wang, Z.1
Zhu, T.2
Qiao, C.3
-
257
-
-
33845218878
-
Gene therapy progress and prospects: Duchenne muscular dystrophy
-
Foster K, Foster H, Dickson JG (2006) Gene therapy progress and prospects: Duchenne muscular dystrophy. Gene Ther 13:1677-1685
-
(2006)
Gene Ther
, vol.13
, pp. 1677-1685
-
-
Foster, K.1
Foster, H.2
Dickson, J.G.3
-
258
-
-
19544378856
-
Haemophilia A: From mutation analysis to new therapies
-
Graw J, Brackmann HH, Oldenburg J et al (2005) Haemophilia A: from mutation analysis to new therapies. Nat Rev Genet 6:488-501
-
(2005)
Nat Rev Genet
, vol.6
, pp. 488-501
-
-
Graw, J.1
Brackmann, H.H.2
Oldenburg, J.3
-
259
-
-
44049090692
-
AAV-mediated gene transfer for the treatment of hemophilia B: Problems and prospects
-
Hasbrouck NC, High KA (2008) AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects. Gene Ther 15:870-875
-
(2008)
Gene Ther
, vol.15
, pp. 870-875
-
-
Hasbrouck, N.C.1
High, K.A.2
-
260
-
-
35349027352
-
Immune responses to AAV in clinical trials
-
Mingozzi F, High KA (2007) Immune responses to AAV in clinical trials. Curr Gene Ther 7:316-324
-
(2007)
Curr Gene Ther
, vol.7
, pp. 316-324
-
-
Mingozzi, F.1
High, K.A.2
-
261
-
-
38949118647
-
Gene therapy for haemophilia
-
Murphy SL, High KA (2008) Gene therapy for haemophilia. Br J Haematol 140:479-487
-
(2008)
Br J Haematol
, vol.140
, pp. 479-487
-
-
Murphy, S.L.1
High, K.A.2
-
262
-
-
33746909909
-
Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B
-
Jiang H, Pierce GF, Ozelo MC et al (2006) Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. Mol Ther 14:452-455
-
(2006)
Mol Ther
, vol.14
, pp. 452-455
-
-
Jiang, H.1
Pierce, G.F.2
Ozelo, M.C.3
-
263
-
-
0034050904
-
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an A AV vector
-
Kay MA, Manno CS, Ragni MV et al (2000) Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an A AV vector. Nat Genet 24:257-261
-
(2000)
Nat Genet
, vol.24
, pp. 257-261
-
-
Kay, M.A.1
Manno, C.S.2
Ragni, M.V.3
-
264
-
-
33644820684
-
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
-
Manno CS, Pierce GF, Arruda VR et al (2006) Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 12:342-347
-
(2006)
Nat Med
, vol.12
, pp. 342-347
-
-
Manno, C.S.1
Pierce, G.F.2
Arruda, V.R.3
-
265
-
-
65549147773
-
Successful treatment of canine hemophilia by continuous expression of canine FVIIa
-
Margaritis P, Roy E, Aljamali MN et al (2009) Successful treatment of canine hemophilia by continuous expression of canine FVIIa. Blood 113:3682-3689
-
(2009)
Blood
, vol.113
, pp. 3682-3689
-
-
Margaritis, P.1
Roy, E.2
Aljamali, M.N.3
-
266
-
-
0034137797
-
Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver
-
Wang L, Nichols TC, Read MS et al (2000) Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver. Mol Ther 1:154-158
-
(2000)
Mol Ther
, vol.1
, pp. 154-158
-
-
Wang, L.1
Nichols, T.C.2
Read, M.S.3
-
267
-
-
0034186366
-
Prodrug activation enzymes in cancer gene therapy
-
Aghi M, Hochberg F, Breakefield XO (2000) Prodrug activation enzymes in cancer gene therapy. J Gene Med 2:148-164
-
(2000)
J Gene Med
, vol.2
, pp. 148-164
-
-
Aghi, M.1
Hochberg, F.2
Breakefield, X.O.3
-
268
-
-
34848903876
-
Plasmid DNA and viral vector-based vaccines for the treatment of cancer
-
Anderson RJ, Schneider J (2007) Plasmid DNA and viral vector-based vaccines for the treatment of cancer. Vaccine 25 [Suppl 2]: B24-34
-
(2007)
Vaccine
, vol.25
, pp. B24-B34
-
-
Anderson, R.J.1
Schneider, J.2
-
269
-
-
45449114994
-
Reprogrammed viruses as cancer therapeutics: Targeted, armed and shielded
-
Cattaneo R, Miest T, Shashkova EV, Barry MA (2008) Reprogrammed viruses as cancer therapeutics: targeted, armed and shielded. Nat Rev Micro 6:529-540
-
(2008)
Nat Rev Micro
, vol.6
, pp. 529-540
-
-
Cattaneo, R.1
Miest, T.2
Shashkova, E.V.3
Barry, M.A.4
-
270
-
-
2942541735
-
Cross-presentation, dendritic cell subsets, and the generation of immunity to cellular antigens
-
Heath WR, Belz GT, Behrens GMN et al (2004) Cross-presentation, dendritic cell subsets, and the generation of immunity to cellular antigens. Immunol Rev 199:9-26
-
(2004)
Immunol Rev
, vol.199
, pp. 9-26
-
-
Heath, W.R.1
Belz, G.T.2
Behrens, G.M.N.3
-
271
-
-
14844352335
-
Genetically based therapeutics for cancer: Similarities and contrasts with traditional drug discovery and development
-
Hermiston TW, Kirn DH (2005) Genetically based therapeutics for cancer: similarities and contrasts with traditional drug discovery and development. Mol Ther 11:496-507
-
(2005)
Mol Ther
, vol.11
, pp. 496-507
-
-
Hermiston, T.W.1
Kirn, D.H.2
-
272
-
-
34249868241
-
Adoptive T cell therapy for cancer in the clinic
-
June CH (2007) Adoptive T cell therapy for cancer in the clinic. J Clin Invest 117:1466-1476
-
(2007)
J Clin Invest
, vol.117
, pp. 1466-1476
-
-
June, C.H.1
-
273
-
-
34248149835
-
Principles of adoptive T cell cancer therapy
-
June CH (2007) Principles of adoptive T cell cancer therapy. J Clin Invest 117:1204-1212
-
(2007)
J Clin Invest
, vol.117
, pp. 1204-1212
-
-
June, C.H.1
-
274
-
-
5444247855
-
Gene transfer approaches in cancer immunotherapy
-
Larin SS, Georgiev GP, Kiselev SL (2004) Gene transfer approaches in cancer immunotherapy. Gene Ther 11 [Suppl 1]: S18-25
-
(2004)
Gene Ther
, vol.11
, pp. S18-S25
-
-
Larin, S.S.1
Georgiev, G.P.2
Kiselev, S.L.3
-
275
-
-
44849135037
-
Gene therapy progress and prospects cancer: Oncolytic viruses
-
Liu TC, Kirn D (2008) Gene therapy progress and prospects cancer: oncolytic viruses. Gene Ther 15:877-884
-
(2008)
Gene Ther
, vol.15
, pp. 877-884
-
-
Liu, T.C.1
Kirn, D.2
-
276
-
-
1842529509
-
Gene therapy progress and prospects: Cancer gene therapy using tumour suppressor genes
-
McNeish IA, Bell SJ, Lemoine NR (2004) Gene therapy progress and prospects: cancer gene therapy using tumour suppressor genes. Gene Ther 11:497-503
-
(2004)
Gene Ther
, vol.11
, pp. 497-503
-
-
McNeish, I.A.1
Bell, S.J.2
Lemoine, N.R.3
-
277
-
-
33749647652
-
Cancer. Cancer immunotherapy is more than a numbers game
-
Offringa R (2006) Cancer. Cancer immunotherapy is more than a numbers game. Science 314:68-69
-
(2006)
Science
, vol.314
, pp. 68-69
-
-
Offringa, R.1
-
279
-
-
38549175830
-
DNA vaccines: Precision tools for activating effective immunity against cancer
-
Rice J, Ottensmeier CH, Stevenson FK (2008) DNA vaccines: precision tools for activating effective immunity against cancer. Nat Rev Cancer 8:108-120
-
(2008)
Nat Rev Cancer
, vol.8
, pp. 108-120
-
-
Rice, J.1
Ottensmeier, C.H.2
Stevenson, F.K.3
-
280
-
-
3042772669
-
Genetic modification of T lymphocytes for adoptive immunotherapy
-
Rossig C, Brenner MK (2004) Genetic modification of T lymphocytes for adoptive immunotherapy. Mol Ther 10:5-18
-
(2004)
Mol Ther
, vol.10
, pp. 5-18
-
-
Rossig, C.1
Brenner, M.K.2
-
281
-
-
0030843493
-
HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia
-
Bonini C, Ferrari G, Verzeletti S et al (1997) HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia. Science 276:1719-1724
-
(1997)
Science
, vol.276
, pp. 1719-1724
-
-
Bonini, C.1
Ferrari, G.2
Verzeletti, S.3
-
282
-
-
34249111217
-
Lentiviral vectors for cancer immunotherapy: Transforming infectious particles into therapeutics
-
Breckpot K, Aerts JL, Thielemans K (2007) Lentiviral vectors for cancer immunotherapy: transforming infectious particles into therapeutics. Gene Ther 14:847-862
-
(2007)
Gene Ther
, vol.14
, pp. 847-862
-
-
Breckpot, K.1
Aerts, J.L.2
Thielemans, K.3
-
283
-
-
44849143678
-
Boosting anti-idiotype immune response with recombinant AAV enhances tumour protection induced by gene gun vaccination
-
Cesco-Gaspere M, Zentilin L, Giacca M, Burrone OR (2008) Boosting anti-idiotype immune response with recombinant AAV enhances tumour protection induced by gene gun vaccination. Scand J Immunol 68:58-66
-
(2008)
Scand J Immunol
, vol.68
, pp. 58-66
-
-
Cesco-Gaspere, M.1
Zentilin, L.2
Giacca, M.3
Burrone, O.R.4
-
284
-
-
34748918373
-
Lessons from randomized phase III studies with active cancer immunotherapies: Outcomes from the 2006 meeting of the Cancer Vaccine Consortium (CVC)
-
Finke LH, Wentworth K, Blumenstein B et al (2007) Lessons from randomized phase III studies with active cancer immunotherapies: outcomes from the 2006 meeting of the Cancer Vaccine Consortium (CVC). Vaccine 25 [Suppl 2]: B97-B109
-
(2007)
Vaccine
, vol.25
, pp. B97-B109
-
-
Finke, L.H.1
Wentworth, K.2
Blumenstein, B.3
-
285
-
-
0024549349
-
Induction of angiogenesis during the transition from hyperplasia to neoplasia
-
Folkman J, Watson K, Ingber D, Hanahan D (1989) Induction of angiogenesis during the transition from hyperplasia to neoplasia. Nature 339:58-61
-
(1989)
Nature
, vol.339
, pp. 58-61
-
-
Folkman, J.1
Watson, K.2
Ingber, D.3
Hanahan, D.4
-
286
-
-
70149114880
-
Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigen
-
Johnson LA, Morgan RA, Dudley ME et al (2009) Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigen. Blood 114:535-546
-
(2009)
Blood
, vol.114
, pp. 535-546
-
-
Johnson, L.A.1
Morgan, R.A.2
Dudley, M.E.3
-
287
-
-
1842529509
-
Gene therapy progress and prospects: Cancer gene therapy using tumour suppressor genes
-
McNeish IA, Bell SJ, Lemoine NR (2004) Gene therapy progress and prospects: cancer gene therapy using tumour suppressor genes. Gene Ther 11:497-503
-
(2004)
Gene Ther
, vol.11
, pp. 497-503
-
-
McNeish, I.A.1
Bell, S.J.2
Lemoine, N.R.3
-
288
-
-
33749624177
-
Cancer regression in patients after transfer of genetically engineered lymphocytes
-
Morgan RA, Dudley ME, Wunderlich JR et al (2006) Cancer regression in patients after transfer of genetically engineered lymphocytes. Science 314:126-129
-
(2006)
Science
, vol.314
, pp. 126-129
-
-
Morgan, R.A.1
Dudley, M.E.2
Wunderlich, J.R.3
-
289
-
-
14844331400
-
Dendritic-cell-based therapeutic vaccination against cancer
-
Nestle FO, Farkas A, Conrad C (2005) Dendritic-cell-based therapeutic vaccination against cancer. Curr Opin Immunol 17:163-169
-
(2005)
Curr Opin Immunol
, vol.17
, pp. 163-169
-
-
Nestle, F.O.1
Farkas, A.2
Conrad, C.3
-
290
-
-
33845336130
-
Characterization of a cancer/testis (CT) antigen gene family capable of eliciting humoral response in cancer patients
-
Parmigiani RB, Bettoni F, Vibranovski MD et al (2006) Characterization of a cancer/testis (CT) antigen gene family capable of eliciting humoral response in cancer patients. Proc Natl Acad Sci U S A 103:18066-18071
-
(2006)
Proc Natl Acad Sci U S A
, vol.103
, pp. 18066-18071
-
-
Parmigiani, R.B.1
Bettoni, F.2
Vibranovski, M.D.3
-
291
-
-
25144436834
-
Gene therapy for malignant glioma: Current clinical status
-
Pulkkanen KJ, Yla-Herttuala S (2005) Gene therapy for malignant glioma: current clinical status. Mol Ther 12:585-598
-
(2005)
Mol Ther
, vol.12
, pp. 585-598
-
-
Pulkkanen, K.J.1
Yla-Herttuala, S.2
-
292
-
-
30744454570
-
Restoration of immunity in lymphopenic individuals with cancer by vaccination and adoptive T-cell transfer
-
Rapoport AP, Stadtmauer EA, Aqui N et al (2005) Restoration of immunity in lymphopenic individuals with cancer by vaccination and adoptive T-cell transfer. Nat Med 11:1230-1237
-
(2005)
Nat Med
, vol.11
, pp. 1230-1237
-
-
Rapoport, A.P.1
Stadtmauer, E.A.2
Aqui, N.3
-
293
-
-
70449516435
-
The allogeneic graft-versus-cancer effect
-
Ringdén O, Karlsson H, Olsson R et al (2009) The allogeneic graft-versus-cancer effect. Br J Haematol 147:614-633
-
(2009)
Br J Haematol
, vol.147
, pp. 614-633
-
-
Ringdén, O.1
Karlsson, H.2
Olsson, R.3
-
294
-
-
0024988334
-
Gene transfer into humans: Immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction
-
Rosenberg SA, Aebersold P, Cornetta K et al (1990) Gene transfer into humans: immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 323:570-578
-
(1990)
N Engl J Med
, vol.323
, pp. 570-578
-
-
Rosenberg, S.A.1
Aebersold, P.2
Cornetta, K.3
-
295
-
-
45849132702
-
Active specific immunotherapy phase III trials for malignant melanoma: Systematic analysis and critical appraisal
-
Rosenthal R, Viehl CT, Guller U et al (2008) Active specific immunotherapy phase III trials for malignant melanoma: systematic analysis and critical appraisal. J Am Coll Surg 207:95-105
-
(2008)
J Am Coll Surg
, vol.207
, pp. 95-105
-
-
Rosenthal, R.1
Viehl, C.T.2
Guller, U.3
-
296
-
-
0037279770
-
Transcellular transfer of active HSV-1 thymidine kinase mediated by an 11-amino-acid peptide from HIV-1 Tat
-
Tasciotti E, Zoppe M, Giacca M (2003) Transcellular transfer of active HSV-1 thymidine kinase mediated by an 11-amino-acid peptide from HIV-1 Tat. Cancer Gene Ther 10:64-74
-
(2003)
Cancer Gene Ther
, vol.10
, pp. 64-74
-
-
Tasciotti, E.1
Zoppe, M.2
Giacca, M.3
-
297
-
-
34748849509
-
Vaccine therapy for melanoma: Current status and future directions
-
Terando AM, Faries MB, Morton DL (2007) Vaccine therapy for melanoma: current status and future directions. Vaccine 25 [Suppl 2]: B4-16
-
(2007)
Vaccine
, vol.25
, pp. B4-B16
-
-
Terando, A.M.1
Faries, M.B.2
Morton, D.L.3
-
298
-
-
33750972592
-
Gene therapy for ALS: Progress and prospects
-
Azzouz M (2006) Gene therapy for ALS: progress and prospects. Biochim Biophys Acta 1762:1122-1127
-
(2006)
Biochim Biophys Acta
, vol.1762
, pp. 1122-1127
-
-
Azzouz, M.1
-
299
-
-
0038823638
-
Gene therapy in autoimmune, demyelinating disease of the central nervous system
-
Baker D, Hankey DJ (2003) Gene therapy in autoimmune, demyelinating disease of the central nervous system. Gene Ther 10:844-853
-
(2003)
Gene Ther
, vol.10
, pp. 844-853
-
-
Baker, D.1
Hankey, D.J.2
-
300
-
-
20444453676
-
Hope for AD with NGF gene-therapy trial
-
Bradbury J (2005) Hope for AD with NGF gene-therapy trial. Lancet Neurol 4:335
-
(2005)
Lancet Neurol
, vol.4
, pp. 335
-
-
Bradbury, J.1
-
301
-
-
0141516387
-
Gene therapy progress and prospects: Parkinson's disease
-
Burton EA, Glorioso JC, Fink DJ (2003) Gene therapy progress and prospects: Parkinson's disease. Gene Ther 10:1721-1727
-
(2003)
Gene Ther
, vol.10
, pp. 1721-1727
-
-
Burton, E.A.1
Glorioso, J.C.2
Fink, D.J.3
-
302
-
-
27844432628
-
Clinical trials in amyotrophic lateral sclerosis: The tenuous past and the promising future
-
Choudry RB, Cudkowicz ME (2005) Clinical trials in amyotrophic lateral sclerosis: the tenuous past and the promising future. J Clin Pharmacol 45:1334-1344
-
(2005)
J Clin Pharmacol
, vol.45
, pp. 1334-1344
-
-
Choudry, R.B.1
Cudkowicz, M.E.2
-
303
-
-
34547805536
-
Gene therapy for peripheral nervous system diseases
-
Federici T, Boulis N (2007) Gene therapy for peripheral nervous system diseases. Curr Gene Ther 7:239-248
-
(2007)
Curr Gene Ther
, vol.7
, pp. 239-248
-
-
Federici, T.1
Boulis, N.2
-
304
-
-
37549059628
-
Current status of gene therapy trials for Parkinson's disease
-
Fiandaca M, Forsayeth J, Bankiewicz K (2008) Current status of gene therapy trials for Parkinson's disease. Exp Neurol 209:51-57
-
(2008)
Exp Neurol
, vol.209
, pp. 51-57
-
-
Fiandaca, M.1
Forsayeth, J.2
Bankiewicz, K.3
-
305
-
-
0042528664
-
Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model
-
Kaspar BK, Llado J, Sherkat N et al (2003) Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science 301:839-842
-
(2003)
Science
, vol.301
, pp. 839-842
-
-
Kaspar, B.K.1
Llado, J.2
Sherkat, N.3
-
306
-
-
34250209501
-
Amyotrophic lateral sclerosis
-
Mitchell JD, Borasio GD (2007) Amyotrophic lateral sclerosis. Lancet 369:2031-2041
-
(2007)
Lancet
, vol.369
, pp. 2031-2041
-
-
Mitchell, J.D.1
Borasio, G.D.2
-
307
-
-
41949128614
-
Towards gene therapy for Parkinson's disease
-
Palfi S (2008) Towards gene therapy for Parkinson's disease. Lancet Neurol 7:375-376
-
(2008)
Lancet Neurol
, vol.7
, pp. 375-376
-
-
Palfi, S.1
-
308
-
-
34547749401
-
Positive clinical data in Parkinson's and ischemia buoy gene therapy
-
Sheridan C (2007) Positive clinical data in Parkinson's and ischemia buoy gene therapy. Nat Biotechnol 25:823-824
-
(2007)
Nat Biotechnol
, vol.25
, pp. 823-824
-
-
Sheridan, C.1
-
309
-
-
33646135323
-
Therapeutics development for spinal muscular atrophy
-
Sumner CJ (2006) Therapeutics development for spinal muscular atrophy. NeuroRx 3:235-245
-
(2006)
NeuroRx
, vol.3
, pp. 235-245
-
-
Sumner, C.J.1
-
310
-
-
0037654372
-
Growth-factor gene therapy for neurodegenerative disorders
-
Tuszynski MH (2002) Growth-factor gene therapy for neurodegenerative disorders. Lancet Neurol 1:51-57
-
(2002)
Lancet Neurol
, vol.1
, pp. 51-57
-
-
Tuszynski, M.H.1
-
311
-
-
70349782901
-
Chapter 20: Gene therapy perspectives for nerve repair
-
Zacchigna S, Giacca M (2009) Chapter 20: Gene therapy perspectives for nerve repair. Int Rev Neurobiol 87:381-392
-
(2009)
Int Rev Neurobiol
, vol.87
, pp. 381-392
-
-
Zacchigna, S.1
Giacca, M.2
-
312
-
-
85047689522
-
Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy
-
Azzouz M, Le T, Ralph GS et al (2004) Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy. J Clin Invest 114:1726-1731
-
(2004)
J Clin Invest
, vol.114
, pp. 1726-1731
-
-
Azzouz, M.1
Le, T.2
Ralph, G.S.3
-
313
-
-
2642526164
-
VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model
-
Azzouz M, Ralph GS, Storkebaum E et al (2004) VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model. Nature 429:413-417
-
(2004)
Nature
, vol.429
, pp. 413-417
-
-
Azzouz, M.1
Ralph, G.S.2
Storkebaum, E.3
-
314
-
-
5644295321
-
Neuroprotective gene therapy for Huntington's disease, using polymer-encapsulated cells engineered to secrete human ciliary neurotrophic factor: Results of a phase I study
-
Bloch J, Bachoud-Levi AC, Deglon N et al (2004) Neuroprotective gene therapy for Huntington's disease, using polymer-encapsulated cells engineered to secrete human ciliary neurotrophic factor: results of a phase I study. Hum Gene Ther 15:968-975
-
(2004)
Hum Gene Ther
, vol.15
, pp. 968-975
-
-
Bloch, J.1
Bachoud-Levi, A.C.2
Deglon, N.3
-
315
-
-
73449128979
-
Safety and tolerability of putaminal AADC gene therapy for Parkinson disease
-
Christine CW, Starr PA, Larson PS et al (2009) Safety and tolerability of putaminal AADC gene therapy for Parkinson disease. Neurology 73:1662-1669
-
(2009)
Neurology
, vol.73
, pp. 1662-1669
-
-
Christine, C.W.1
Starr, P.A.2
Larson, P.S.3
-
316
-
-
44949222522
-
Results from a phase I safety trial of hAADC gene therapy for Parkinson disease
-
Eberling JL, Jagust WJ, Christine CW et al (2008) Results from a phase I safety trial of hAADC gene therapy for Parkinson disease. Neurology 70:1980-1983
-
(2008)
Neurology
, vol.70
, pp. 1980-1983
-
-
Eberling, J.L.1
Jagust, W.J.2
Christine, C.W.3
-
317
-
-
41549168514
-
Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice
-
Hua Y, Vickers TA, Okunola HL et al (2008) Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice. Am J Hum Genet 82:834-848
-
(2008)
Am J Hum Genet
, vol.82
, pp. 834-848
-
-
Hua, Y.1
Vickers, T.A.2
Okunola, H.L.3
-
318
-
-
34250683023
-
Safety and tolerability of gene therapy with an adenoassociated virus (AAV) borne GAD gene for Parkinson's disease: An open label, phase I trial
-
Kaplitt MG, Feigin A, Tang C et al (2007) Safety and tolerability of gene therapy with an adenoassociated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 369:2097-2105
-
(2007)
Lancet
, vol.369
, pp. 2097-2105
-
-
Kaplitt, M.G.1
Feigin, A.2
Tang, C.3
-
319
-
-
65249117415
-
Gene therapy delivers an alternative approach to Alzheimer's disease
-
Kennington E (2009) Gene therapy delivers an alternative approach to Alzheimer's disease. Nat Rev Drug Discov 8:275
-
(2009)
Nat Rev Drug Discov
, vol.8
, pp. 275
-
-
Kennington, E.1
-
320
-
-
37549042441
-
Design of clinical trials of gene therapy in Parkinson disease
-
Lewis TB, Standaert DG (2008) Design of clinical trials of gene therapy in Parkinson disease. Exp Neurol 209:41-47
-
(2008)
Exp Neurol
, vol.209
, pp. 41-47
-
-
Lewis, T.B.1
Standaert, D.G.2
-
321
-
-
6344283047
-
Clinical trials in neurological disorders using A AV vectors: Promises and challenges
-
Mandel RJ, Burger C (2004) Clinical trials in neurological disorders using A AV vectors: promises and challenges. Curr Opin Mol Ther 6:482-490
-
(2004)
Curr Opin Mol Ther
, vol.6
, pp. 482-490
-
-
Mandel, R.J.1
Burger, C.2
-
322
-
-
37549054118
-
Viral vectors for in vivo gene transfer in Parkinson's disease: Properties and clinical grade production
-
Mandel RJ, Burger C, Snyder RO (2008) Viral vectors for in vivo gene transfer in Parkinson's disease: properties and clinical grade production. Exp Neurol 209:58-71
-
(2008)
Exp Neurol
, vol.209
, pp. 58-71
-
-
Mandel, R.J.1
Burger, C.2
Snyder, R.O.3
-
323
-
-
62049083420
-
Neuroprotective effects of brain-derived neurotrophic factor in rodent and primate models of Alzheimer's disease
-
Nagahara AH, Merrill DA, Coppola G et al (2009) Neuroprotective effects of brain-derived neurotrophic factor in rodent and primate models of Alzheimer's disease. Nat Med 15:331-337
-
(2009)
Nat Med
, vol.15
, pp. 331-337
-
-
Nagahara, A.H.1
Merrill, D.A.2
Coppola, G.3
-
324
-
-
20244381261
-
Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model
-
Ralph GS, Radcliffe PA, Day DM et al (2005) Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model. Nat Med 11:429-433
-
(2005)
Nat Med
, vol.11
, pp. 429-433
-
-
Ralph, G.S.1
Radcliffe, P.A.2
Day, D.M.3
-
325
-
-
4644234505
-
VEGF: Once regarded as a specific angiogenic factor, now implicated in neuroprotection
-
Storkebaum E, Lambrechts D, Carmeliet P (2004) VEGF: once regarded as a specific angiogenic factor, now implicated in neuroprotection. Bioessays 26:943-954
-
(2004)
Bioessays
, vol.26
, pp. 943-954
-
-
Storkebaum, E.1
Lambrechts, D.2
Carmeliet, P.3
-
326
-
-
74349088517
-
Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6
-
Towne C, Schneider BL, Kieran D et al (2009) Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6. Gene Ther 17:141-146
-
(2009)
Gene Ther
, vol.17
, pp. 141-146
-
-
Towne, C.1
Schneider, B.L.2
Kieran, D.3
-
327
-
-
21044458854
-
A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer disease
-
Tuszynski MH, Thal L, Pay M et al (2005) A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer disease. Nat Med 11:551-555
-
(2005)
Nat Med
, vol.11
, pp. 551-555
-
-
Tuszynski, M.H.1
Thal, L.2
Pay, M.3
-
328
-
-
50049136340
-
Success in sight: The eyes have it! Ocular gene therapy trials for LCA look promising
-
Bainbridge JW, Ali RR (2008) Success in sight: the eyes have it! Ocular gene therapy trials for LCA look promising. Gene Ther 15:1191-1192
-
(2008)
Gene Ther
, vol.15
, pp. 1191-1192
-
-
Bainbridge, J.W.1
Ali, R.R.2
-
329
-
-
33746863749
-
Gene therapy progress and prospects: The eye
-
Bainbridge JW, Tan MH, Ali RR (2006) Gene therapy progress and prospects: the eye. Gene Ther 13:1191-1197
-
(2006)
Gene Ther
, vol.13
, pp. 1191-1197
-
-
Bainbridge, J.W.1
Tan, M.H.2
Ali, R.R.3
-
330
-
-
0034200932
-
Gene therapy for ocular disease
-
Bennett J, Maguire AM (2000) Gene therapy for ocular disease. Mol Ther 1:501-505
-
(2000)
Mol Ther
, vol.1
, pp. 501-505
-
-
Bennett, J.1
Maguire, A.M.2
-
331
-
-
44049100684
-
AAV-mediated gene therapy for retinal disorders: From mouse to man
-
Buch PK, Bainbridge JW, Ali RR (2008) AAV-mediated gene therapy for retinal disorders: from mouse to man. Gene Ther 15:849-857
-
(2008)
Gene Ther
, vol.15
, pp. 849-857
-
-
Buch, P.K.1
Bainbridge, J.W.2
Ali, R.R.3
-
333
-
-
43249113220
-
Gene therapy. Two teams report progress in reversing loss of sight
-
Kaiser J (2008) Gene therapy. Two teams report progress in reversing loss of sight. Science 320:606-607
-
(2008)
Science
, vol.320
, pp. 606-607
-
-
Kaiser, J.1
-
335
-
-
0035032662
-
Gene therapy restores vision in a canine model of childhood blindness
-
Acland GM, Aguirre GD, Ray J et al (2001) Gene therapy restores vision in a canine model of childhood blindness. Nat Genet 28:92-95
-
(2001)
Nat Genet
, vol.28
, pp. 92-95
-
-
Acland, G.M.1
Aguirre, G.D.2
Ray, J.3
-
336
-
-
44249120315
-
Effect of gene therapy on visual function in Leber's congenital amaurosis
-
Bainbridge JW, Smith AJ, Barker SS et al (2008) Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 358:2231-2239
-
(2008)
N Engl J Med
, vol.358
, pp. 2231-2239
-
-
Bainbridge, J.W.1
Smith, A.J.2
Barker, S.S.3
-
338
-
-
33751103471
-
Age-related macular degeneration: Emerging pathogenetic and therapeutic concepts
-
Gehrs KM, Anderson DH, Johnson LV, Hageman GS (2006) Age-related macular degeneration: emerging pathogenetic and therapeutic concepts. Ann Med 38:450-471
-
(2006)
Ann Med
, vol.38
, pp. 450-471
-
-
Gehrs, K.M.1
Anderson, D.H.2
Johnson, L.V.3
Hageman, G.S.4
-
339
-
-
33846933945
-
Restoration of vision in RPE65-deficient Briard dogs using an A AV serotype 4 vector that specifically targets the retinal pigmented epithelium
-
Le Meur G, Stieger K, Smith AJ et al (2007) Restoration of vision in RPE65-deficient Briard dogs using an A AV serotype 4 vector that specifically targets the retinal pigmented epithelium. Gene Ther 14:292-303
-
(2007)
Gene Ther
, vol.14
, pp. 292-303
-
-
Le Meur, G.1
Stieger, K.2
Smith, A.J.3
-
340
-
-
44249085878
-
Safety and efficacy of gene transfer for Leber's congenital amaurosis
-
Maguire AM, Simonelli F, Pierce EA et al (2008) Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 358:2240-2248
-
(2008)
N Engl J Med
, vol.358
, pp. 2240-2248
-
-
Maguire, A.M.1
Simonelli, F.2
Pierce, E.A.3
-
341
-
-
34249689753
-
Molecular regulation of angiogenesis and lymphangiogenesis
-
Adams RH, Alitalo K (2007) Molecular regulation of angiogenesis and lymphangiogenesis. Nat Rev Mol Cell Biol 8:464-478
-
(2007)
Nat Rev Mol Cell Biol
, vol.8
, pp. 464-478
-
-
Adams, R.H.1
Alitalo, K.2
-
342
-
-
60749096085
-
Control of vascular morphogenesis and homeostasis through the angiopoietin-Tie system
-
Augustin HG, Koh G Y, Thurston G, Alitalo K (2009) Control of vascular morphogenesis and homeostasis through the angiopoietin-Tie system. Nat Rev Mol Cell Biol 10:165-177
-
(2009)
Nat Rev Mol Cell Biol
, vol.10
, pp. 165-177
-
-
Augustin, H.G.1
Koh, G.Y.2
Thurston, G.3
Alitalo, K.4
-
344
-
-
30744479430
-
Angiogenesis in life, disease and medicine
-
Carmeliet P (2005) Angiogenesis in life, disease and medicine. Nature 438:932-936
-
(2005)
Nature
, vol.438
, pp. 932-936
-
-
Carmeliet, P.1
-
345
-
-
0037385683
-
Gene transfer strategies to inhibit neointima formation
-
Crook MF, Akyurek LM (2003) Gene transfer strategies to inhibit neointima formation. Trends Cardiovasc Med 13:102-106
-
(2003)
Trends Cardiovasc Med
, vol.13
, pp. 102-106
-
-
Crook, M.F.1
Akyurek, L.M.2
-
346
-
-
70349281951
-
Virus-mediated gene transfer to induce therapeutic angiogenesis: Where do we stand?
-
Giacca M (2007) Virus-mediated gene transfer to induce therapeutic angiogenesis: where do we stand? Int J Nanomed 2:527-540
-
(2007)
Int J Nanomed
, vol.2
, pp. 527-540
-
-
Giacca, M.1
-
348
-
-
34250772826
-
Current status of cardiovascular gene therapy
-
Rissanen TT, Yla-Herttuala S (2007) Current status of cardiovascular gene therapy. Mol Ther 15:1233-1247
-
(2007)
Mol Ther
, vol.15
, pp. 1233-1247
-
-
Rissanen, T.T.1
Yla-Herttuala, S.2
-
349
-
-
0034820506
-
More 'malignant' than cancer? Five-year survival following a first admission for heart failure
-
Stewart S, MacIntyre K, Hole DJ et al (2001) More 'malignant' than cancer? Five-year survival following a first admission for heart failure. Eur J Heart Fail 3:315-322
-
(2001)
Eur J Heart Fail
, vol.3
, pp. 315-322
-
-
Stewart, S.1
MacIntyre, K.2
Hole, D.J.3
-
350
-
-
34248155707
-
Gene therapy progress and prospects: Therapeutic angiogenesis for ischemic cardiovascular disease
-
Vincent KA, Jiang C, Boltje I, Kelly RA (2007) Gene therapy progress and prospects: therapeutic angiogenesis for ischemic cardiovascular disease. Gene Ther 14:781-789
-
(2007)
Gene Ther
, vol.14
, pp. 781-789
-
-
Vincent, K.A.1
Jiang, C.2
Boltje, I.3
Kelly, R.A.4
-
352
-
-
0034648793
-
Vascular-specific growth factors and blood vessel formation
-
Yancopoulos GD, Davis S, Gale NW et al (2000) Vascular-specific growth factors and blood vessel formation. Nature 407:242-248
-
(2000)
Nature
, vol.407
, pp. 242-248
-
-
Yancopoulos, G.D.1
Davis, S.2
Gale, N.W.3
-
353
-
-
0037968240
-
Gene transfer as a tool to induce therapeutic vascular growth
-
Yla-Herttuala S, Alitalo K (2003) Gene transfer as a tool to induce therapeutic vascular growth. Nat Med 9:694-701
-
(2003)
Nat Med
, vol.9
, pp. 694-701
-
-
Yla-Herttuala, S.1
Alitalo, K.2
-
354
-
-
18844417604
-
Gene therapy for ischemic cardiovascular diseases: Some lessons learned from the first clinical trials
-
Yla-Herttuala S, Markkanen JE, Rissanen TT (2004) Gene therapy for ischemic cardiovascular diseases: some lessons learned from the first clinical trials. Trends Cardiovasc Med 14:295-300
-
(2004)
Trends Cardiovasc Med
, vol.14
, pp. 295-300
-
-
Yla-Herttuala, S.1
Markkanen, J.E.2
Rissanen, T.T.3
-
355
-
-
0034650348
-
Cardiovascular gene therapy
-
Yla-Herttuala S, Martin JF (2000) Cardiovascular gene therapy. Lancet 355:213-222
-
(2000)
Lancet
, vol.355
, pp. 213-222
-
-
Yla-Herttuala, S.1
Martin, J.F.2
-
356
-
-
7044235846
-
Vascular endothelial growth factor stimulates skeletal muscle regeneration in vivo
-
Arsic N, Zacchigna S, Zentilin L et al (2004) Vascular endothelial growth factor stimulates skeletal muscle regeneration in vivo. Mol Ther 10:844-854
-
(2004)
Mol Ther
, vol.10
, pp. 844-854
-
-
Arsic, N.1
Zacchigna, S.2
Zentilin, L.3
-
357
-
-
0037699954
-
The biology of VEGF and its receptors
-
Arsic N, Zentilin L, Zacchigna S et al (2003) The biology of VEGF and its receptors. Nat Med 9:669-676
-
(2003)
Nat Med
, vol.9
, pp. 669-676
-
-
Arsic, N.1
Zentilin, L.2
Zacchigna, S.3
-
358
-
-
0032583951
-
Constitutive expression of phVEGF165 after intramuscular gene transfer promotes collateral vessel development in patients with critical limb ischemia
-
Baumgartner I, Pieczek A, Manor O et al (1998) Constitutive expression of phVEGF165 after intramuscular gene transfer promotes collateral vessel development in patients with critical limb ischemia. Circulation 97:1114-1123
-
(1998)
Circulation
, vol.97
, pp. 1114-1123
-
-
Baumgartner, I.1
Pieczek, A.2
Manor, O.3
-
359
-
-
0033534053
-
Restoration of contractile function in isolated cardiomyocytes from failing human hearts by gene transfer of SERCA2a
-
Del Monte F, Harding SE, Schmidt U et al (1999) Restoration of contractile function in isolated cardiomyocytes from failing human hearts by gene transfer of SERCA2a. Circulation 100:2308-2311
-
(1999)
Circulation
, vol.100
, pp. 2308-2311
-
-
Del Monte, F.1
Harding, S.E.2
Schmidt, U.3
-
360
-
-
33646128955
-
Adeno-associated virus-mediated transduction of VEGF165 improves cardiac tissue viability and functional recovery after permanent coronary occlusion in conscious dogs
-
Ferrarini M, Arsic N, Recchia FA et al (2006) Adeno-associated virus-mediated transduction of VEGF165 improves cardiac tissue viability and functional recovery after permanent coronary occlusion in conscious dogs. Circ Res 98:954-961
-
(2006)
Circ Res
, vol.98
, pp. 954-961
-
-
Ferrarini, M.1
Arsic, N.2
Recchia, F.A.3
-
361
-
-
0037133624
-
Angiogenic Gene Therapy (AGENT) trial in patients with stable angina pectoris
-
Grines CL, Watkins MW, Helmer G et al (2002) Angiogenic Gene Therapy (AGENT) trial in patients with stable angina pectoris. Circulation 105:1291-1297
-
(2002)
Circulation
, vol.105
, pp. 1291-1297
-
-
Grines, C.L.1
Watkins, M.W.2
Helmer, G.3
-
362
-
-
33745143956
-
Robust systemic transduction with AAV9 vectors in mice: Efficient global cardiac gene transfer superior to that of AAV8
-
Inagaki K, Fuess S, Storm TA et al (2006) Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol Ther 14:45-53
-
(2006)
Mol Ther
, vol.14
, pp. 45-53
-
-
Inagaki, K.1
Fuess, S.2
Storm, T.A.3
-
363
-
-
0028792587
-
Which gene for which restenosis?
-
Lafont A, Guerot C, Lemarchand P (1995) Which gene for which restenosis? Lancet 346:1442-1443
-
(1995)
Lancet
, vol.346
, pp. 1442-1443
-
-
Lafont, A.1
Guerot, C.2
Lemarchand, P.3
-
364
-
-
1842559592
-
Gene and cell-based therapies for heart disease
-
Melo LG, Pachori AS, Kong D et al (2004) Gene and cell-based therapies for heart disease. FASEB J 18:648-663
-
(2004)
FASEB J
, vol.18
, pp. 648-663
-
-
Melo, L.G.1
Pachori, A.S.2
Kong, D.3
-
365
-
-
0034641624
-
Clinical trials in coronary angiogenesis: Issues, problems, consensus: An expert panel summary
-
Simons M, Bonow RO, Chronos NA et al (2000) Clinical trials in coronary angiogenesis: issues, problems, consensus: an expert panel summary. Circulation 102:E73-E86
-
(2000)
Circulation
, vol.102
, pp. E73-E86
-
-
Simons, M.1
Bonow, R.O.2
Chronos, N.A.3
-
366
-
-
0037689525
-
Induction of functional neovascularization by combined VEGF and angiopoietin-1 gene transfer using AAV vectors
-
Sinagra G, Giacca M (2003) Induction of functional neovascularization by combined VEGF and angiopoietin-1 gene transfer using AAV vectors. Mol Ther 7:450-459
-
(2003)
Mol Ther
, vol.7
, pp. 450-459
-
-
Sinagra, G.1
Giacca, M.2
-
367
-
-
68949163249
-
Inducible adeno-associated virus vectors promote functional angiogenesis in adult organisms via regulated vascular endothelial growth factor expression
-
Tafuro S, Ayuso E, Zacchigna S et al (2009) Inducible adeno-associated virus vectors promote functional angiogenesis in adult organisms via regulated vascular endothelial growth factor expression. Cardiovasc Res 83:663-671
-
(2009)
Cardiovasc Res
, vol.83
, pp. 663-671
-
-
Tafuro, S.1
Ayuso, E.2
Zacchigna, S.3
-
368
-
-
0034730094
-
Left ventricular electromechanical mapping to assess efficacy of phVEGF (165) gene transfer for therapeutic angiogenesis in chronic myocardial ischemia
-
Vale PR, Losordo DW, Milliken CE et al (2000) Left ventricular electromechanical mapping to assess efficacy of phVEGF (165) gene transfer for therapeutic angiogenesis in chronic myocardial ischemia. Circulation 102:965-974
-
(2000)
Circulation
, vol.102
, pp. 965-974
-
-
Vale, P.R.1
Losordo, D.W.2
Milliken, C.E.3
-
369
-
-
34447126305
-
In vivo imaging shows abnormal function of vascular endothelial growth factor-induced vasculature
-
Zacchigna S, Tasciotti E, Kusmic C et al (2007) In vivo imaging shows abnormal function of vascular endothelial growth factor-induced vasculature. Hum Gene Ther 18:515-524
-
(2007)
Hum Gene Ther
, vol.18
, pp. 515-524
-
-
Zacchigna, S.1
Tasciotti, E.2
Kusmic, C.3
-
370
-
-
0024299563
-
Gene therapy. Intracellular immunization
-
Baltimore D (1988) Gene therapy. Intracellular immunization. Nature 335:395-396
-
(1988)
Nature
, vol.335
, pp. 395-396
-
-
Baltimore, D.1
-
371
-
-
33745646047
-
Gene-based immunotherapy for human immunodeficiency virus infection and acquired immunodeficiency syndrome
-
Dropulic B, June CH (2006) Gene-based immunotherapy for human immunodeficiency virus infection and acquired immunodeficiency syndrome. Hum Gene Ther 17:577-588
-
(2006)
Hum Gene Ther
, vol.17
, pp. 577-588
-
-
Dropulic, B.1
June, C.H.2
-
372
-
-
0037832252
-
Suicide gene therapy mediated by the Herpes Simplex virus thymidine kinase gene/Ganciclovir system: Fifteen years of application
-
Fillat C, Carrio M, Cascante A, Sangro B (2003) Suicide gene therapy mediated by the Herpes Simplex virus thymidine kinase gene/Ganciclovir system: fifteen years of application. Curr Gene Ther 3:13-26
-
(2003)
Curr Gene Ther
, vol.3
, pp. 13-26
-
-
Fillat, C.1
Carrio, M.2
Cascante, A.3
Sangro, B.4
-
373
-
-
37249086660
-
Gene therapy to induce cellular resistance to HIV-1 infection: Lessons from clinical trials
-
Giacca M (2008) Gene therapy to induce cellular resistance to HIV-1 infection: lessons from clinical trials. Adv Pharmacol 56:297-325
-
(2008)
Adv Pharmacol
, vol.56
, pp. 297-325
-
-
Giacca, M.1
-
374
-
-
36849042601
-
RNA interference against viruses: Strike and counterstrike
-
Haasnoot J, Westerhout EM, Berkhout B (2007) RNA interference against viruses: strike and counterstrike. Nat Biotechnol 25:1435-1443
-
(2007)
Nat Biotechnol
, vol.25
, pp. 1435-1443
-
-
Haasnoot, J.1
Westerhout, E.M.2
Berkhout, B.3
-
375
-
-
20844455902
-
Regulatory considerations for novel gene therapy products: A review of the process leading to the first clinical lentiviral vector
-
Manilla P, Rebello T, Afable C et al (2005) Regulatory considerations for novel gene therapy products: a review of the process leading to the first clinical lentiviral vector. Hum Gene Ther 16:17-25
-
(2005)
Hum Gene Ther
, vol.16
, pp. 17-25
-
-
Manilla, P.1
Rebello, T.2
Afable, C.3
-
376
-
-
33645144814
-
Lentiviral-mediated delivery of siRNAs for antiviral therapy
-
Morris K. V, Rossi JJ (2006) Lentiviral-mediated delivery of siRNAs for antiviral therapy. Gene Ther 13:553-558
-
(2006)
Gene Ther
, vol.13
, pp. 553-558
-
-
Morris, K.V.1
Rossi, J.J.2
-
377
-
-
33646848909
-
RNAi as a treatment for HIV-1 infection
-
Rossi JJ (2006) RNAi as a treatment for HIV-1 infection. Biotechniques [Suppl]: 25-29
-
(2006)
Biotechniques
, pp. 25-29
-
-
Rossi, J.J.1
-
379
-
-
19444370090
-
Current status of gene therapy strategies to treat HIV/AIDS
-
Strayer DS, Akkina R, Bunnell BA et al (2005) Current status of gene therapy strategies to treat HIV/AIDS. Mol Ther 11:823-842
-
(2005)
Mol Ther
, vol.11
, pp. 823-842
-
-
Strayer, D.S.1
Akkina, R.2
Bunnell, B.A.3
-
380
-
-
15944391296
-
Gene therapy progress and prospects: Novel gene therapy approaches for AIDS
-
Wolkowicz R, Nolan GP (2005) Gene therapy progress and prospects: novel gene therapy approaches for AIDS. Gene Ther 12:467-476
-
(2005)
Gene Ther
, vol.12
, pp. 467-476
-
-
Wolkowicz, R.1
Nolan, G.P.2
-
381
-
-
56649105122
-
Efficacy assessment of a cell-mediated immunity HIV-1 vaccine (the Step Study): A double-blind, randomised, placebo-controlled, test-ofconcept trial
-
Buchbinder SP, Mehrotra D V, Duerr A et al (2008) Efficacy assessment of a cell-mediated immunity HIV-1 vaccine (the Step Study): a double-blind, randomised, placebo-controlled, test-ofconcept trial. Lancet 372:1881-1893
-
(2008)
Lancet
, vol.372
, pp. 1881-1893
-
-
Buchbinder, S.P.1
Mehrotra, D.V.2
Duerr, A.3
-
382
-
-
1242319384
-
Human immunodeficiency virus type 1 escapes from RNA interference-mediated inhibition
-
Das AT, Brummelkamp TR, Westerhout EM et al (2004) Human immunodeficiency virus type 1 escapes from RNA interference-mediated inhibition. J Virol 78:2601-2605
-
(2004)
J Virol
, vol.78
, pp. 2601-2605
-
-
Das, A.T.1
Brummelkamp, T.R.2
Westerhout, E.M.3
-
383
-
-
33751251058
-
Gene transfer in humans using a conditionally replicating lentiviral vector
-
Levine BL, Humeau LM, Boyer J et al (2006) Gene transfer in humans using a conditionally replicating lentiviral vector. Proc Natl Acad Sci U S A 103:17372-17377
-
(2006)
Proc Natl Acad Sci U S A
, vol.103
, pp. 17372-17377
-
-
Levine, B.L.1
Humeau, L.M.2
Boyer, J.3
-
384
-
-
27744605569
-
Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy
-
Li MJ, Kim J, Li S et al (2005) Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy. Mol Ther 12:900-909
-
(2005)
Mol Ther
, vol.12
, pp. 900-909
-
-
Li, M.J.1
Kim, J.2
Li, S.3
-
385
-
-
62049083710
-
Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells
-
Mitsuyasu RT, Merigan TC, Carr A et al (2009) Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells. Nat Med 15:285-292
-
(2009)
Nat Med
, vol.15
, pp. 285-292
-
-
Mitsuyasu, R.T.1
Merigan, T.C.2
Carr, A.3
-
386
-
-
0030594579
-
Gene therapy for AIDS using retroviral mediated gene transfer to deliver HIV-1 antisense TAR and transdominant Rev protein genes to syngeneic lymphocytes in HIV-1 infected identical twins
-
Morgan RA, Walker R (1996) Gene therapy for AIDS using retroviral mediated gene transfer to deliver HIV-1 antisense TAR and transdominant Rev protein genes to syngeneic lymphocytes in HIV-1 infected identical twins. Hum Gene Ther 7:1281-1306
-
(1996)
Hum Gene Ther
, vol.7
, pp. 1281-1306
-
-
Morgan, R.A.1
Walker, R.2
-
387
-
-
27244444559
-
TRIM family proteins: Retroviral restriction and antiviral defence
-
Nisole S, Stoye JP, Saib A (2005) TRIM family proteins: retroviral restriction and antiviral defence. Nat Rev Microbiol 3:799-808
-
(2005)
Nat Rev Microbiol
, vol.3
, pp. 799-808
-
-
Nisole, S.1
Stoye, J.P.2
Saib, A.3
-
388
-
-
0036066508
-
SiRNA-directed inhibition of HIV-1 infection
-
Novina CD, Murray MF, Dykxhoorn DM et al (2002) siRNA-directed inhibition of HIV-1 infection. Nat Med 8:681-686
-
(2002)
Nat Med
, vol.8
, pp. 681-686
-
-
Novina, C.D.1
Murray, M.F.2
Dykxhoorn, D.M.3
-
390
-
-
0030818332
-
Intracellular antibodies (intrabodies) for gene therapy of infectious diseases
-
Rondon IJ, Marasco WA (1997) Intracellular antibodies (intrabodies) for gene therapy of infectious diseases. Annu Rev Microbiol 51:257-283
-
(1997)
Annu Rev Microbiol
, vol.51
, pp. 257-283
-
-
Rondon, I.J.1
Marasco, W.A.2
-
391
-
-
0025363891
-
Ribozymes as potential anti-HIV-1 therapeutic agents
-
Sarver N, Cantin EM, Chang PS et al (1990) Ribozymes as potential anti-HIV-1 therapeutic agents. Science 247:1222-1225
-
(1990)
Science
, vol.247
, pp. 1222-1225
-
-
Sarver, N.1
Cantin, E.M.2
Chang, P.S.3
-
392
-
-
33745219190
-
Towards hematopoietic stem cell-mediated protection against infection with human immunodeficiency virus
-
Schambach A, Schiedlmeier B, Kuhlcke K et al (2006) Towards hematopoietic stem cell-mediated protection against infection with human immunodeficiency virus. Gene Ther 13:1037-1047
-
(2006)
Gene Ther
, vol.13
, pp. 1037-1047
-
-
Schambach, A.1
Schiedlmeier, B.2
Kuhlcke, K.3
-
393
-
-
33751001588
-
Silencing of HIV-1 with RNA interference: A multiple shRNA approach
-
Ter Brake O, Konstantinova P, Ceylan M, Berkhout B (2006) Silencing of HIV-1 with RNA interference: a multiple shRNA approach. Mol Ther 14:883-892
-
(2006)
Mol Ther
, vol.14
, pp. 883-892
-
-
Ter Brake, O.1
Konstantinova, P.2
Ceylan, M.3
Berkhout, B.4
-
394
-
-
13844320402
-
HIV-1 can escape from RNA interference by evolving an alternative structure in its RNA genome
-
Westerhout EM, Ooms M, Vink M et al (2005) HIV-1 can escape from RNA interference by evolving an alternative structure in its RNA genome. Nucleic Acids Res 33:796-804
-
(2005)
Nucleic Acids Res
, vol.33
, pp. 796-804
-
-
Westerhout, E.M.1
Ooms, M.2
Vink, M.3
-
395
-
-
23844466665
-
Gene therapy for lipoprotein disorders
-
Broedl UC, Rader DJ (2005) Gene therapy for lipoprotein disorders. Exp Opin Biol Ther 5:1029-1038
-
(2005)
Exp Opin Biol Ther
, vol.5
, pp. 1029-1038
-
-
Broedl, U.C.1
Rader, D.J.2
-
396
-
-
5444237545
-
Liver gene therapy: Advances and hurdles
-
Nguyen TH, Ferry N (2004) Liver gene therapy: advances and hurdles. Gene Ther 11 [Suppl 1]: S76-84
-
(2004)
Gene Ther
, vol.11
, pp. S76-S84
-
-
Nguyen, T.H.1
Ferry, N.2
-
397
-
-
65949115488
-
Ex vivo liver-directed gene therapy for the treatment of metabolic diseases: Advances in hepatocyte transplantation and retroviral vectors
-
Nguyen TH, Mainot S, Lainas P et al (2009) Ex vivo liver-directed gene therapy for the treatment of metabolic diseases: advances in hepatocyte transplantation and retroviral vectors. Curr Gene Ther 9:136-149
-
(2009)
Curr Gene Ther
, vol.9
, pp. 136-149
-
-
Nguyen, T.H.1
Mainot, S.2
Lainas, P.3
-
398
-
-
0037271797
-
Gene therapy progress and prospects: Alpha-1 antitrypsin
-
Stecenko AA, Brigham KL (2003) Gene therapy progress and prospects: alpha-1 antitrypsin. Gene Ther 10:95-99
-
(2003)
Gene Ther
, vol.10
, pp. 95-99
-
-
Stecenko, A.A.1
Brigham, K.L.2
-
399
-
-
34547647784
-
Alpha one antitrypsin deficiency: From gene to treatment
-
Wood AM, Stockley RA (2007) Alpha one antitrypsin deficiency: from gene to treatment. Respiration 74:481-492
-
(2007)
Respiration
, vol.74
, pp. 481-492
-
-
Wood, A.M.1
Stockley, R.A.2
-
400
-
-
0028793489
-
A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia
-
Grossman M, Rader DJ, Muller DW et al (1995) A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia. Nat Med 1:1148-1154
-
(1995)
Nat Med
, vol.1
, pp. 1148-1154
-
-
Grossman, M.1
Rader, D.J.2
Muller, D.W.3
-
401
-
-
0028292602
-
Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia
-
Grossman M, Raper SE, Kozarsky K et al (1994) Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia. Nat Genet 6:335-341
-
(1994)
Nat Genet
, vol.6
, pp. 335-341
-
-
Grossman, M.1
Raper, S.E.2
Kozarsky, K.3
-
402
-
-
0029988465
-
Effective treatment of familial hypercholester - Olaemia in the mouse model using adenovirus-mediated transfer of the VLDL receptor gene
-
Kozarsky KF, Jooss K, Donahee M et al (1996) Effective treatment of familial hypercholester - olaemia in the mouse model using adenovirus-mediated transfer of the VLDL receptor gene. Nat Genet 13:54-62
-
(1996)
Nat Genet
, vol.13
, pp. 54-62
-
-
Kozarsky, K.F.1
Jooss, K.2
Donahee, M.3
-
403
-
-
65949095203
-
Towards liver-directed gene therapy for Crigler-Najjar syndrome
-
Miranda PS, Bosma PJ (2009) Towards liver-directed gene therapy for Crigler-Najjar syndrome. Curr Gene Ther 9:72-82
-
(2009)
Curr Gene Ther
, vol.9
, pp. 72-82
-
-
Miranda, P.S.1
Bosma, P.J.2
-
404
-
-
20744450475
-
Alpha1-antitrypsin deficiency
-
Stoller JK, Aboussouan LS (2005) Alpha1-antitrypsin deficiency. Lancet 365:2225-2236
-
(2005)
Lancet
, vol.365
, pp. 2225-2236
-
-
Stoller, J.K.1
Aboussouan, L.S.2
-
405
-
-
38649136193
-
If it's broken, shouldn't it be fixed? Informed consent and initial clinical trials of gene therapy
-
Caplan AL (2008) If it's broken, shouldn't it be fixed? Informed consent and initial clinical trials of gene therapy. Hum Gene Ther 19:5-6
-
(2008)
Hum Gene Ther
, vol.19
, pp. 5-6
-
-
Caplan, A.L.1
-
406
-
-
34249656800
-
Cognitive regeneration or enhancement: The ethical issues
-
Chan S, Harris J (2006) Cognitive regeneration or enhancement: the ethical issues. Regen Med 1:361-366
-
(2006)
Regen Med
, vol.1
, pp. 361-366
-
-
Chan, S.1
Harris, J.2
-
408
-
-
27944470542
-
Gene therapy progress and prospects: Fetal gene therapy - First proofs of concept - Some adverse effects
-
Coutelle C, Themis M, Waddington SN et al (2005) Gene therapy progress and prospects: fetal gene therapy - first proofs of concept - some adverse effects. Gene Ther 12:1601-1607
-
(2005)
Gene Ther
, vol.12
, pp. 1601-1607
-
-
Coutelle, C.1
Themis, M.2
Waddington, S.N.3
-
409
-
-
70449527808
-
Accepting risk in clinical research: Is the gene therapy field becoming too risk-averse?
-
Deakin CT, Alexander IE, Kerridge I (2009) Accepting risk in clinical research: is the gene therapy field becoming too risk-averse? Mol Ther 17:1842-1848
-
(2009)
Mol Ther
, vol.17
, pp. 1842-1848
-
-
Deakin, C.T.1
Alexander, I.E.2
Kerridge, I.3
-
410
-
-
39449134828
-
Enhancement is good for you!: Understanding the ethics of genetic enhancement
-
Harris J, Chan S (2008) Enhancement is good for you!: understanding the ethics of genetic enhancement. Gene Ther 15:338-339
-
(2008)
Gene Ther
, vol.15
, pp. 338-339
-
-
Harris, J.1
Chan, S.2
-
411
-
-
38649123009
-
Informed consent in human gene transfer clinical trials
-
Kahn J (2008) Informed consent in human gene transfer clinical trials. Hum Gene Ther 19:7-8
-
(2008)
Hum Gene Ther
, vol.19
, pp. 7-8
-
-
Kahn, J.1
-
412
-
-
39449132142
-
Progress and prospects: Gene therapy for performance and appearance enhancement
-
Kiuru M, Crystal RG (2008) Progress and prospects: gene therapy for performance and appearance enhancement. Gene Ther 15:329-337
-
(2008)
Gene Ther
, vol.15
, pp. 329-337
-
-
Kiuru, M.1
Crystal, R.G.2
-
413
-
-
8844266041
-
Gene therapy progress and prospects: Bringing gene therapy into medical practice: The evolution of international ethics and the regulatory environment
-
Spink J, Geddes D (2004) Gene therapy progress and prospects: bringing gene therapy into medical practice: the evolution of international ethics and the regulatory environment. Gene Ther 11:1611-1616
-
(2004)
Gene Ther
, vol.11
, pp. 1611-1616
-
-
Spink, J.1
Geddes, D.2
-
414
-
-
44349148405
-
Gene doping: The hype and the reality
-
Wells DJ (2008) Gene doping: the hype and the reality. Br J Pharmacol 154:623-631
-
(2008)
Br J Pharmacol
, vol.154
, pp. 623-631
-
-
Wells, D.J.1
-
415
-
-
0033588888
-
Recombinant adenovirus gene transfer in adults with partial ornithine transcarbamylase deficiency (OTCD)
-
Batshaw ML, Wilson JM, Raper S et al (1999) Recombinant adenovirus gene transfer in adults with partial ornithine transcarbamylase deficiency (OTCD). Hum Gene Ther 10:2419-2437
-
(1999)
Hum Gene Ther
, vol.10
, pp. 2419-2437
-
-
Batshaw, M.L.1
Wilson, J.M.2
Raper, S.3
-
416
-
-
67650497832
-
Investigation of the cause of death in a gene-therapy trial
-
Frank KM, Hogarth DK, Miller JL et al (2009) Investigation of the cause of death in a gene-therapy trial. N Engl J Med 361:161-169
-
(2009)
N Engl J Med
, vol.361
, pp. 161-169
-
-
Frank, K.M.1
Hogarth, D.K.2
Miller, J.L.3
-
417
-
-
33745299723
-
Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways
-
Grimm D, Streetz KL, Jopling CL et al (2006) Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 441:537-541
-
(2006)
Nature
, vol.441
, pp. 537-541
-
-
Grimm, D.1
Streetz, K.L.2
Jopling, C.L.3
-
418
-
-
34548045718
-
Clinical research. Death prompts a review of gene therapy vector
-
Kaiser J (2007) Clinical research. Death prompts a review of gene therapy vector. Science 317:580
-
(2007)
Science
, vol.317
, pp. 580
-
-
Kaiser, J.1
-
419
-
-
40949120900
-
Full-length dystrophin gene transfer to the mdx mouse in utero
-
Reay DP, Bilbao R, Koppanati BM et al (2008) Full-length dystrophin gene transfer to the mdx mouse in utero. Gene Ther 15:531-536
-
(2008)
Gene Ther
, vol.15
, pp. 531-536
-
-
Reay, D.P.1
Bilbao, R.2
Koppanati, B.M.3
|