-
1
-
-
0034724857
-
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
-
Cavazzana-Calvo, M.; Hacein-Bey, S.; Basile, C. D.; Gross, F.; Yvon, E.; Nusbaum, P.; Selz, F.; Hue, C.; Certain, S.; Casanova, J. L.; Bousso, P.; Le Deist, F.; Fischer, A. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease Science 2000, 288 (5466) 669-672
-
(2000)
Science
, vol.288
, Issue.5466
, pp. 669-672
-
-
Cavazzana-Calvo, M.1
Hacein-Bey, S.2
Basile, C.D.3
Gross, F.4
Yvon, E.5
Nusbaum, P.6
Selz, F.7
Hue, C.8
Certain, S.9
Casanova, J.L.10
Bousso, P.11
Le Deist, F.12
Fischer, A.13
-
2
-
-
78650922808
-
Gene therapy of chronic granulomatous disease: The engraftment dilemma
-
Grez, M.; Reichenbach, J.; Schwable, J.; Seger, R.; Dinauer, M. C.; Thrasher, A. J. Gene therapy of chronic granulomatous disease: The engraftment dilemma Mol. Ther. 2011, 19 (1) 28-35
-
(2011)
Mol. Ther.
, vol.19
, Issue.1
, pp. 28-35
-
-
Grez, M.1
Reichenbach, J.2
Schwable, J.3
Seger, R.4
Dinauer, M.C.5
Thrasher, A.J.6
-
3
-
-
0035822038
-
Medical progress - The hemophilias - From royal genes to gene therapy
-
Mannucci, P. M.; Tuddenham, E. G. D. Medical progress-The hemophilias-From royal genes to gene therapy N. Engl. J. Med. 2001, 344 (23) 1773-1779
-
(2001)
N. Engl. J. Med.
, vol.344
, Issue.23
, pp. 1773-1779
-
-
Mannucci, P.M.1
Tuddenham, E.G.D.2
-
4
-
-
0347365764
-
An overview of current delivery systems in cancer gene therapy
-
El-Aneed, A. An overview of current delivery systems in cancer gene therapy J. Controlled Release 2004, 94 (1) 1-14
-
(2004)
J. Controlled Release
, vol.94
, Issue.1
, pp. 1-14
-
-
El-Aneed, A.1
-
5
-
-
34250683023
-
Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: An open label, phase i trial
-
Kaplitt, M. G.; Feigin, A.; Tang, C.; Fitzsimons, H. L.; Mattis, P.; Lawlor, P. A.; Bland, R. J.; Young, D.; Strybing, K.; Eidelberg, D.; During, M. J. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial Lancet 2007, 369 (9579) 2097-2105
-
(2007)
Lancet
, vol.369
, Issue.9579
, pp. 2097-2105
-
-
Kaplitt, M.G.1
Feigin, A.2
Tang, C.3
Fitzsimons, H.L.4
Mattis, P.5
Lawlor, P.A.6
Bland, R.J.7
Young, D.8
Strybing, K.9
Eidelberg, D.10
During, M.J.11
-
6
-
-
0037309716
-
Barriers to nonviral gene delivery
-
Wiethoff, C. M.; Middaugh, C. R. Barriers to nonviral gene delivery J. Pharm. Sci. 2003, 92 (2) 203-217
-
(2003)
J. Pharm. Sci.
, vol.92
, Issue.2
, pp. 203-217
-
-
Wiethoff, C.M.1
Middaugh, C.R.2
-
7
-
-
0030670810
-
Toxicity and immunomodulatory activity of liposomal vectors formulated with cationic lipids toward immune effector cells
-
Filion, M. C.; Phillips, N. C. Toxicity and immunomodulatory activity of liposomal vectors formulated with cationic lipids toward immune effector cells Biochim. Biophys. Acta, Biomembr. 1997, 1329 (2) 345-356
-
(1997)
Biochim. Biophys. Acta, Biomembr.
, vol.1329
, Issue.2
, pp. 345-356
-
-
Filion, M.C.1
Phillips, N.C.2
-
8
-
-
0025934603
-
Interaction of positively-charged liposomes with blood - Implications for their application in vivo
-
Senior, J. H.; Trimble, K. R.; Maskiewicz, R. Interaction of positively-charged liposomes with blood-Implications for their application in vivo Biochim. Biophys. Acta 1991, 1070 (1) 173-179
-
(1991)
Biochim. Biophys. Acta
, vol.1070
, Issue.1
, pp. 173-179
-
-
Senior, J.H.1
Trimble, K.R.2
Maskiewicz, R.3
-
9
-
-
13844298046
-
Intracellular delivery of large molecules and small particles by cell-penetrating proteins and peptides
-
Gupta, B.; Levchenko, T. S.; Torchilin, V. P. Intracellular delivery of large molecules and small particles by cell-penetrating proteins and peptides Adv. Drug Delivery Rev. 2005, 57 (4) 637-651
-
(2005)
Adv. Drug Delivery Rev.
, vol.57
, Issue.4
, pp. 637-651
-
-
Gupta, B.1
Levchenko, T.S.2
Torchilin, V.P.3
-
10
-
-
0035053829
-
Ligand-receptor-mediated drug delivery: An emerging paradigm in cellular drug targeting
-
Vyas, S. P.; Singh, A.; Sihorkar, V. Ligand-receptor-mediated drug delivery: an emerging paradigm in cellular drug targeting Crit. Rev. Ther. Drug Carrier Syst. 2001, 18 (1) 1-76
-
(2001)
Crit. Rev. Ther. Drug Carrier Syst.
, vol.18
, Issue.1
, pp. 1-76
-
-
Vyas, S.P.1
Singh, A.2
Sihorkar, V.3
-
11
-
-
0030928619
-
Gene therapy - Promises, problems and prospects
-
Verma, I. M.; Somia, N. Gene therapy-promises, problems and prospects Nature 1997, 389 (6648) 239-242
-
(1997)
Nature
, vol.389
, Issue.6648
, pp. 239-242
-
-
Verma, I.M.1
Somia, N.2
-
12
-
-
64549134676
-
Nonviral vectors for gene delivery
-
Mintzer, M. A.; Simanek, E. E. Nonviral vectors for gene delivery Chem. Rev. 2008, 109 (2) 259-302
-
(2008)
Chem. Rev.
, vol.109
, Issue.2
, pp. 259-302
-
-
Mintzer, M.A.1
Simanek, E.E.2
-
13
-
-
73449101112
-
Recent trends in non-viral vector-mediated gene delivery
-
Pathak, A.; Patnaik, S.; Gupta, K. C. Recent trends in non-viral vector-mediated gene delivery Biotechnol. J. 2009, 4 (11) 1559-1572
-
(2009)
Biotechnol. J.
, vol.4
, Issue.11
, pp. 1559-1572
-
-
Pathak, A.1
Patnaik, S.2
Gupta, K.C.3
-
14
-
-
60949104907
-
Polymeric materials for gene delivery and DNA vaccination
-
Nguyen, D. N.; Green, J. J.; Chan, J. M.; Langer, R.; Anderson, D. G. Polymeric materials for gene delivery and DNA vaccination Adv. Mater. 2009, 21 (8) 847-867
-
(2009)
Adv. Mater.
, vol.21
, Issue.8
, pp. 847-867
-
-
Nguyen, D.N.1
Green, J.J.2
Chan, J.M.3
Langer, R.4
Anderson, D.G.5
-
15
-
-
84884816169
-
Poly(ethylene glycol)- block -cationic polylactide nanocomplexes of differing charge density for gene delivery
-
Chen, C. K.; Jones, C. H.; Mistriotis, P.; Yu, Y.; Ma, X. N.; Ravikrishnan, A.; Jiang, M.; Andreadis, S. T.; Pfeifer, B. A.; Cheng, C. Poly(ethylene glycol)- block -cationic polylactide nanocomplexes of differing charge density for gene delivery Biomaterials 2013, 34 (37) 9688-9699
-
(2013)
Biomaterials
, vol.34
, Issue.37
, pp. 9688-9699
-
-
Chen, C.K.1
Jones, C.H.2
Mistriotis, P.3
Yu, Y.4
Ma, X.N.5
Ravikrishnan, A.6
Jiang, M.7
Andreadis, S.T.8
Pfeifer, B.A.9
Cheng, C.10
-
16
-
-
0141681128
-
Synthesis of poly(beta-amino ester)s optimized for highly effective gene delivery
-
Akinc, A.; Anderson, D. G.; Lynn, D. M.; Langer, R. Synthesis of poly(beta-amino ester)s optimized for highly effective gene delivery Bioconjugate Chem. 2003, 14 (5) 979-988
-
(2003)
Bioconjugate Chem.
, vol.14
, Issue.5
, pp. 979-988
-
-
Akinc, A.1
Anderson, D.G.2
Lynn, D.M.3
Langer, R.4
-
17
-
-
33746438424
-
Toxicity of cationic lipids and cationic polymers in gene delivery
-
Lv, H.; Zhang, S.; Wang, B.; Cui, S.; Yan, J. Toxicity of cationic lipids and cationic polymers in gene delivery J. Controlled Release 2006, 114 (1) 100-109
-
(2006)
J. Controlled Release
, vol.114
, Issue.1
, pp. 100-109
-
-
Lv, H.1
Zhang, S.2
Wang, B.3
Cui, S.4
Yan, J.5
-
18
-
-
70649098228
-
Tissue-specific gene delivery via nanoparticle coating
-
Harris, T. J.; Green, J. J.; Fung, P. W.; Langer, R.; Anderson, D. G.; Bhatia, S. N. Tissue-specific gene delivery via nanoparticle coating Biomaterials 2010, 31 (5) 998-1006
-
(2010)
Biomaterials
, vol.31
, Issue.5
, pp. 998-1006
-
-
Harris, T.J.1
Green, J.J.2
Fung, P.W.3
Langer, R.4
Anderson, D.G.5
Bhatia, S.N.6
-
19
-
-
33847136300
-
Cationic polysaccharides for gene delivery
-
Yudovin-Farber, I.; Domb, A. J. Cationic polysaccharides for gene delivery Mater. Sci. Eng., C 2007, 27 (3) 595-598
-
(2007)
Mater. Sci. Eng., C
, vol.27
, Issue.3
, pp. 595-598
-
-
Yudovin-Farber, I.1
Domb, A.J.2
-
20
-
-
33847192134
-
Peptide-guided gene delivery
-
Martin, M.; Rice, K. Peptide-guided gene delivery AAPS J. 2007, 9 (1) E18-E29
-
(2007)
AAPS J.
, vol.9
, Issue.1
-
-
Martin, M.1
Rice, K.2
-
21
-
-
0034786764
-
Chitosan as a nonviral gene delivery system. Structure-property relationships and characteristics compared with polyethylenimine in vitro and after lung administration in vivo
-
Koping-Hoggard, M.; Tubulekas, I.; Guan, H.; Edwards, K.; Nilsson, M.; Varum, K. M.; Artursson, P. Chitosan as a nonviral gene delivery system. Structure-property relationships and characteristics compared with polyethylenimine in vitro and after lung administration in vivo Gene Ther. 2001, 8 (14) 1108-1121
-
(2001)
Gene Ther.
, vol.8
, Issue.14
, pp. 1108-1121
-
-
Koping-Hoggard, M.1
Tubulekas, I.2
Guan, H.3
Edwards, K.4
Nilsson, M.5
Varum, K.M.6
Artursson, P.7
-
22
-
-
0032958073
-
Oral gene delivery with chitosan-DNA nanoparticles generates immunologic protection in a murine model of peanut allergy
-
Roy, K.; Mao, H. Q.; Huang, S. K.; Leong, K. W. Oral gene delivery with chitosan-DNA nanoparticles generates immunologic protection in a murine model of peanut allergy Nat. Med. 1999, 5 (4) 387-391
-
(1999)
Nat. Med.
, vol.5
, Issue.4
, pp. 387-391
-
-
Roy, K.1
Mao, H.Q.2
Huang, S.K.3
Leong, K.W.4
-
23
-
-
0037103242
-
Arginine-rich molecular transporters for drug delivery: Role of backbone spacing in cellular uptake
-
Rothbard, J. B.; Kreider, E.; Vandeusen, C. L.; Wright, L.; Wylie, B. L.; Wender, P. A. Arginine-rich molecular transporters for drug delivery: Role of backbone spacing in cellular uptake J. Med. Chem. 2002, 45 (17) 3612-3618
-
(2002)
J. Med. Chem.
, vol.45
, Issue.17
, pp. 3612-3618
-
-
Rothbard, J.B.1
Kreider, E.2
Vandeusen, C.L.3
Wright, L.4
Wylie, B.L.5
Wender, P.A.6
-
25
-
-
0037592877
-
Insight into the mechanism of the peptide-based gene delivery system MPG: Implications for delivery of siRNA into mammalian cells
-
Simeoni, F.; Morris, M. C.; Heitz, F.; Divita, G. Insight into the mechanism of the peptide-based gene delivery system MPG: implications for delivery of siRNA into mammalian cells Nucleic Acids Res. 2003, 31 (11) 2717-2724
-
(2003)
Nucleic Acids Res.
, vol.31
, Issue.11
, pp. 2717-2724
-
-
Simeoni, F.1
Morris, M.C.2
Heitz, F.3
Divita, G.4
-
26
-
-
67349279624
-
Peptide-mediated DNA condensation for non-viral gene therapy
-
Saccardo, P.; Villaverde, A.; Gonzalez-Montalban, N. Peptide-mediated DNA condensation for non-viral gene therapy Biotechnol. Adv. 2009, 27 (4) 432-438
-
(2009)
Biotechnol. Adv.
, vol.27
, Issue.4
, pp. 432-438
-
-
Saccardo, P.1
Villaverde, A.2
Gonzalez-Montalban, N.3
-
27
-
-
0042927082
-
Comparison between the interactions of adenovirus-derived peptides with plasmid DNA and their role in gene delivery mediated by liposome-peptide-DNA virus-like nanoparticles
-
Preuss, M.; Tecle, M.; Shah, I.; Matthews, D. A.; Miller, A. D. Comparison between the interactions of adenovirus-derived peptides with plasmid DNA and their role in gene delivery mediated by liposome-peptide-DNA virus-like nanoparticles Org. Biomol. Chem. 2003, 1 (14) 2430-2438
-
(2003)
Org. Biomol. Chem.
, vol.1
, Issue.14
, pp. 2430-2438
-
-
Preuss, M.1
Tecle, M.2
Shah, I.3
Matthews, D.A.4
Miller, A.D.5
-
28
-
-
0035803784
-
Histidine-rich peptides and polymers for nucleic acids delivery
-
Pichon, C.; Goncalves, C.; Midoux, P. Histidine-rich peptides and polymers for nucleic acids delivery Adv. Drug Delivery Rev. 2001, 53 (1) 75-94
-
(2001)
Adv. Drug Delivery Rev.
, vol.53
, Issue.1
, pp. 75-94
-
-
Pichon, C.1
Goncalves, C.2
Midoux, P.3
-
29
-
-
68549130637
-
Chemical vectors for gene delivery: A current review on polymers, peptides and lipids containing histidine or imidazole as nucleic acids carriers
-
Midoux, P.; Pichon, C.; Yaouanc, J.-J.; Jaffrès, P.-A. Chemical vectors for gene delivery: a current review on polymers, peptides and lipids containing histidine or imidazole as nucleic acids carriers Br. J. Pharmacol. 2009, 157 (2) 166-178
-
(2009)
Br. J. Pharmacol.
, vol.157
, Issue.2
, pp. 166-178
-
-
Midoux, P.1
Pichon, C.2
Yaouanc, J.-J.3
Jaffrès, P.-A.4
-
30
-
-
84869387296
-
Selective gene delivery to cancer cells using an integrated cationic amphiphilic peptide
-
Tang, Q.; Cao, B.; Wu, H. Y.; Cheng, G. Selective gene delivery to cancer cells using an integrated cationic amphiphilic peptide Langmuir 2012, 28 (46) 16126-16132
-
(2012)
Langmuir
, vol.28
, Issue.46
, pp. 16126-16132
-
-
Tang, Q.1
Cao, B.2
Wu, H.Y.3
Cheng, G.4
-
31
-
-
84859944275
-
Dextran conjugates in drug delivery
-
Varshosaz, J. Dextran conjugates in drug delivery Expert Opin. Drug Delivery 2012, 9 (5) 509-523
-
(2012)
Expert Opin. Drug Delivery
, vol.9
, Issue.5
, pp. 509-523
-
-
Varshosaz, J.1
-
32
-
-
84858745745
-
Optimized dextran-polyethylenimine conjugates are efficient non-viral vectors with reduced cytotoxicity when used in serum containing environments
-
Jiang, D. H.; Salem, A. K. Optimized dextran-polyethylenimine conjugates are efficient non-viral vectors with reduced cytotoxicity when used in serum containing environments Int. J. Pharm. 2012, 427 (1) 71-79
-
(2012)
Int. J. Pharm.
, vol.427
, Issue.1
, pp. 71-79
-
-
Jiang, D.H.1
Salem, A.K.2
-
33
-
-
84873881095
-
Cholesterol-peptide hybrids to form liposome-like vesicles for gene delivery
-
Tang, Q.; Cao, B.; Wu, H. Y.; Cheng, G. Cholesterol-peptide hybrids to form liposome-like vesicles for gene delivery. PLoS One 2013, 8 (1).
-
(2013)
PLoS One
, vol.8
, Issue.1
-
-
Tang, Q.1
Cao, B.2
Wu, H.Y.3
Cheng, G.4
-
34
-
-
0031548721
-
Reaction of dextran with glycidyl methacrylate: An unexpected transesterification
-
vanDijkWolthuis, W. N. E.; KettenesvandenBosch, J. J.; vanderKerkvanHoof, A.; Hennink, W. E. Reaction of dextran with glycidyl methacrylate: An unexpected transesterification Macromolecules 1997, 30 (11) 3411-3413
-
(1997)
Macromolecules
, vol.30
, Issue.11
, pp. 3411-3413
-
-
Vandijkwolthuis, W.N.E.1
Kettenesvandenbosch, J.J.2
Vanderkerkvanhoof, A.3
Hennink, W.E.4
-
35
-
-
84896844840
-
Zwitteration of dextran: A facile route to integrate antifouling, switchability and optical transparency into natural polymers
-
10.1039/c3cc48878k
-
Cao, B.; Li, L. L.; Wu, H. Y.; Qiong, T.; Sun, B. B.; Dong, H.; Zhe, J.; Cheng, G. Zwitteration of dextran: a facile route to integrate antifouling, switchability and optical transparency into natural polymers Chem. Commun. 2014, 10.1039/c3cc48878k
-
(2014)
Chem. Commun.
-
-
Cao, B.1
Li, L.L.2
Wu, H.Y.3
Qiong, T.4
Sun, B.B.5
Dong, H.6
Zhe, J.7
Cheng, G.8
-
36
-
-
33646679654
-
Michael addition reactions in macromolecular design for emerging technologies
-
Mather, B. D.; Viswanathan, K.; Miller, K. M.; Long, T. E. Michael addition reactions in macromolecular design for emerging technologies Prog. Polym. Sci. 2006, 31 (5) 487-531
-
(2006)
Prog. Polym. Sci.
, vol.31
, Issue.5
, pp. 487-531
-
-
Mather, B.D.1
Viswanathan, K.2
Miller, K.M.3
Long, T.E.4
-
37
-
-
84892145853
-
Co-delivery of small interfering RNA using a camptothecin prodrug as the carrier
-
Tang, Q.; Cao, B.; Cheng, G. Co-delivery of small interfering RNA using a camptothecin prodrug as the carrier Chem. Commun. 2013, 50 (11) 1323-1325
-
(2013)
Chem. Commun.
, vol.50
, Issue.11
, pp. 1323-1325
-
-
Tang, Q.1
Cao, B.2
Cheng, G.3
-
38
-
-
41549140043
-
Self-assembled cationic peptide nanoparticles capable of inducing efficient gene expression in vitro
-
Wiradharma, N.; Khan, M.; Tong, Y. W.; Wang, S.; Yang, Y. Y. Self-assembled cationic peptide nanoparticles capable of inducing efficient gene expression in vitro Adv. Funct. Mater. 2008, 18 (6) 943-951
-
(2008)
Adv. Funct. Mater.
, vol.18
, Issue.6
, pp. 943-951
-
-
Wiradharma, N.1
Khan, M.2
Tong, Y.W.3
Wang, S.4
Yang, Y.Y.5
-
39
-
-
0034605974
-
Examination of the biophysical interaction between plasmid DNA and the polycations, polylysine and polyornithine, as a basis for their differential gene transfection in-vitro
-
Ramsay, E.; Hadgraft, J.; Birchall, J.; Gumbleton, M. Examination of the biophysical interaction between plasmid DNA and the polycations, polylysine and polyornithine, as a basis for their differential gene transfection in-vitro Int. J. Pharm. 2000, 210 (1-2) 97-107
-
(2000)
Int. J. Pharm.
, vol.210
, Issue.12
, pp. 97-107
-
-
Ramsay, E.1
Hadgraft, J.2
Birchall, J.3
Gumbleton, M.4
-
40
-
-
0035980982
-
Design and gene delivery activity of modified polyethylenimines
-
Kircheis, R.; Wightman, L.; Wagner, E. Design and gene delivery activity of modified polyethylenimines Adv. Drug Delivery Rev. 2001, 53 (3) 341-358
-
(2001)
Adv. Drug Delivery Rev.
, vol.53
, Issue.3
, pp. 341-358
-
-
Kircheis, R.1
Wightman, L.2
Wagner, E.3
-
41
-
-
0346634895
-
In vitro cytotoxicity testing of polycations: Influence of polymer structure on cell viability and hemolysis
-
Fischer, D.; Li, Y.; Ahlemeyer, B.; Krieglstein, J.; Kissel, T. In vitro cytotoxicity testing of polycations: influence of polymer structure on cell viability and hemolysis Biomaterials 2003, 24 (7) 1121-1131
-
(2003)
Biomaterials
, vol.24
, Issue.7
, pp. 1121-1131
-
-
Fischer, D.1
Li, Y.2
Ahlemeyer, B.3
Krieglstein, J.4
Kissel, T.5
-
42
-
-
0024355622
-
Biochemical characterisation of polycation-induced cytotoxicity to human vascular endothelial cells
-
Morgan, D. M.; Larvin, V. L.; Pearson, J. D. Biochemical characterisation of polycation-induced cytotoxicity to human vascular endothelial cells J. Cell Sci. 1989, 94 (3) 553-559
-
(1989)
J. Cell Sci.
, vol.94
, Issue.3
, pp. 553-559
-
-
Morgan, D.M.1
Larvin, V.L.2
Pearson, J.D.3
-
43
-
-
0038004448
-
A novel non-viral vector for DNA delivery based on low molecular weight, branched polyethylenimine: Effect of molecular weight on transfection efficiency and cytotoxicity
-
Fischer, D.; Bieber, T.; Li, Y. X.; Elsasser, H. P.; Kissel, T. A novel non-viral vector for DNA delivery based on low molecular weight, branched polyethylenimine: Effect of molecular weight on transfection efficiency and cytotoxicity Pharm. Res. 1999, 16 (8) 1273-1279
-
(1999)
Pharm. Res.
, vol.16
, Issue.8
, pp. 1273-1279
-
-
Fischer, D.1
Bieber, T.2
Li, Y.X.3
Elsasser, H.P.4
Kissel, T.5
|