-
1
-
-
21244491577
-
AAV hybrid serotypes: Improved vectors for gene delivery
-
Choi VW, McCarty DM, Samulski RJ: AAV hybrid serotypes: improved vectors for gene delivery. Curr Gene Ther 2005; 5: 299-310.
-
(2005)
Curr Gene Ther
, vol.5
, pp. 299-310
-
-
Choi, V.W.1
McCarty, D.M.2
Samulski, R.J.3
-
2
-
-
0029741735
-
Safety of single-dose administration of an adeno-associated virus (AAV)- CFTR vector in the primate lung
-
Conrad CK, Allen SS, Afione SA, Reynolds TC, Beck SE, Fee-Maki M, Barrazza-Ortiz X, Adams R, Askin FB, Carter BJ, Guggino WB, Flotte TR: Safety of single-dose administration of an adeno-associated virus (AAV)- CFTR vector in the primate lung. Gene Ther 1996; 3: 658-668.
-
(1996)
Gene Ther
, vol.3
, pp. 658-668
-
-
Conrad, C.K.1
Allen, S.S.2
Afione, S.A.3
Reynolds, T.C.4
Beck, S.E.5
Fee-Maki, M.6
Barrazza-Ortiz, X.7
Adams, R.8
Askin, F.B.9
Carter, B.J.10
Guggino, W.B.11
Flotte, T.R.12
-
3
-
-
0031875751
-
In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector
-
During MJ, Samulski RJ, Elsworth JD, Kaplitt MG, Leone P, Xiao X, Li J, Freese A, Taylor JR, Roth RH, Sladek JR Jr, O'Malley KL, Redmond DE Jr: In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector. Gene Ther 1998; 5: 820-827.
-
(1998)
Gene Ther
, vol.5
, pp. 820-827
-
-
During, M.J.1
Samulski, R.J.2
Elsworth, J.D.3
Kaplitt, M.G.4
Leone, P.5
Xiao, X.6
Li, J.7
Freese, A.8
Taylor, J.R.9
Roth, R.H.10
Sladek Jr., J.R.11
O'Malley, K.L.12
Redmond Jr., D.E.13
-
4
-
-
2642512201
-
Clades of adenoassociated viruses are widely disseminated in human tissues
-
Gao G, Vandenberghe LH, Alvira MR, Lu Y, Calcedo R, Zhou X, Wilson JM: Clades of adenoassociated viruses are widely disseminated in human tissues. J Virol 2004; 78: 6381-6388.
-
(2004)
J Virol
, vol.78
, pp. 6381-6388
-
-
Gao, G.1
Vandenberghe, L.H.2
Alvira, M.R.3
Lu, Y.4
Calcedo, R.5
Zhou, X.6
Wilson, J.M.7
-
6
-
-
0037015049
-
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
-
Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J, Wilson JM: Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 2002; 99: 11854-11859.
-
(2002)
Proc Natl Acad Sci USA
, vol.99
, pp. 11854-11859
-
-
Gao, G.P.1
Alvira, M.R.2
Wang, L.3
Calcedo, R.4
Johnston, J.5
Wilson, J.M.6
-
7
-
-
0034963928
-
Incorporation of tumor-targeting peptides into recombinant adeno-associated virus capsids
-
Grifman M, Trepel M, Speece P, Gilbert LB, Arap W, Pasqualini R, Weitzman MD: Incorporation of tumor-targeting peptides into recombinant adeno-associated virus capsids. Mol Ther 2001; 3: 964-975.
-
(2001)
Mol Ther
, vol.3
, pp. 964-975
-
-
Grifman, M.1
Trepel, M.2
Speece, P.3
Gilbert, L.B.4
Arap, W.5
Pasqualini, R.6
Weitzman, M.D.7
-
8
-
-
0038150506
-
Helper virus-free, optically controllable, and twoplasmid- based production of adeno-associated virus vectors of serotypes 1 to 6
-
Grimm D, Kay MA, Kleinschmidt JA: Helper virus-free, optically controllable, and twoplasmid- based production of adeno-associated virus vectors of serotypes 1 to 6. Mol Ther 2003; 7: 839-850.
-
(2003)
Mol Ther
, vol.7
, pp. 839-850
-
-
Grimm, D.1
Kay, M.A.2
Kleinschmidt, J.A.3
-
9
-
-
0032506752
-
Novel tools for production and purification of recombinant adenoassociated virus vectors
-
Grimm D, Kern A, Rittner K, Kleinschmidt JA: Novel tools for production and purification of recombinant adenoassociated virus vectors. Hum Gene Ther 1998; 9: 2745-2760.
-
(1998)
Hum Gene Ther
, vol.9
, pp. 2745-2760
-
-
Grimm, D.1
Kern, A.2
Rittner, K.3
Kleinschmidt, J.A.4
-
11
-
-
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 MG, Feigin A, Tang C, Fitzsimons HL, Mattis P, Lawlor PA, Bland RJ, Young D, Strybing K, Eidelberg D, During MJ: 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: 2097-2105.
-
(2007)
Lancet
, vol.369
, 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
-
12
-
-
1842527048
-
Identification of adeno-associated virus contamination in cell and virus stocks by PCR
-
Katano H, Afione S, Schmidt M, Chiorini JA: Identification of adeno-associated virus contamination in cell and virus stocks by PCR. BioTechniques 2004; 36: 676-680.
-
(2004)
BioTechniques
, vol.36
, pp. 676-680
-
-
Katano, H.1
Afione, S.2
Schmidt, M.3
Chiorini, J.A.4
-
13
-
-
33846593292
-
Development of AAV serotype-specific ELISAs using novel monoclonal antibodies
-
Kuck D, Kern A, Kleinschmidt JA: Development of AAV serotype-specific ELISAs using novel monoclonal antibodies. J Virol Methods 2007; 140: 17-24.
-
(2007)
J Virol Methods
, vol.140
, pp. 17-24
-
-
Kuck, D.1
Kern, A.2
Kleinschmidt, J.A.3
-
14
-
-
0041375553
-
Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver
-
Loiler SA, Conlon TJ, Song S, Tang Q, Warrington KH, Agarwal A, Kapturczak M, Li C, Ricordi C, Atkinson MA, Muzyczka N, Flotte TR: Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver. Gene Ther 2003; 10: 1551-1558.
-
(2003)
Gene Ther
, vol.10
, pp. 1551-1558
-
-
Loiler, S.A.1
Conlon, T.J.2
Song, S.3
Tang, Q.4
Warrington, K.H.5
Agarwal, A.6
Kapturczak, M.7
Li, C.8
Ricordi, C.9
Atkinson, M.A.10
Muzyczka, N.11
Flotte, T.R.12
-
15
-
-
37549054118
-
Viral vectors for in vivo gene transfer in Parkinson's disease: Properties and clinical grade production
-
Mandel RJ, Burger C, Snyder RO: Viral vectors for in vivo gene transfer in Parkinson's disease: properties and clinical grade production. Exp Neurol 2008; 209: 58-71.
-
(2008)
Exp Neurol
, vol.209
, pp. 58-71
-
-
Mandel, R.J.1
Burger, C.2
Snyder, R.O.3
-
16
-
-
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, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA: Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response. Nat Med 2006; 12: 342-347.
-
(2006)
Nat Med
, vol.12
, pp. 342-347
-
-
Manno, C.S.1
Pierce, G.F.2
Arruda, V.R.3
Glader, B.4
Ragni, M.5
Rasko, J.J.6
Ozelo, M.C.7
Hoots, K.8
Blatt, P.9
Konkle, B.10
Dake, M.11
Kaye, R.12
Razavi, M.13
Zajko, A.14
Zehnder, J.15
Rustagi, P.K.16
Nakai, H.17
Chew, A.18
Leonard, D.19
Wright, J.F.20
Lessard, R.R.21
Sommer, J.M.22
Tigges, M.23
Sabatino, D.24
Luk, A.25
Jiang, H.26
Mingozzi, F.27
Couto, L.28
Ertl, H.C.29
High, K.A.30
Kay, M.A.31
more..
-
17
-
-
0031875874
-
Adeno-associated virus vectors can be efficiently produced without helper virus
-
Matsushita T, Elliger S, Elliger C, Podsakoff G, Villarreal L, Kurtzman GJ, Iwaki Y, Colosi P: Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther 1998; 5: 938-945.
-
(1998)
Gene Ther
, vol.5
, pp. 938-945
-
-
Matsushita, T.1
Elliger, S.2
Elliger, C.3
Podsakoff, G.4
Villarreal, L.5
Kurtzman, G.J.6
Iwaki, Y.7
Colosi, P.8
-
18
-
-
33745889596
-
Serotype-specific detection of adenoassociated virus during laboratory preparation
-
Mitchell DA, O'Donnell J, Hare JT, Chapman MS: Serotype-specific detection of adenoassociated virus during laboratory preparation. J Virol Methods 2006; 136: 277-282.
-
(2006)
J Virol Methods
, vol.136
, pp. 277-282
-
-
Mitchell, D.A.1
O'Donnell, J.2
Hare, J.T.3
Chapman, M.S.4
-
19
-
-
9644265332
-
Two novel adeno-associated viruses from cynomolgus monkey: Pseudotyping characterization of capsid protein
-
Mori S, Wang L, Takeuchi T, Kanda T: Two novel adeno-associated viruses from cynomolgus monkey: pseudotyping characterization of capsid protein. Virology 2004; 330: 375-383.
-
(2004)
Virology
, vol.330
, pp. 375-383
-
-
Mori, S.1
Wang, L.2
Takeuchi, T.3
Kanda, T.4
-
20
-
-
0042360384
-
Random peptide libraries displayed on adenoassociated virus to select for targeted gene therapy vectors
-
Muller OJ, Kaul F, Weitzman MD, Pasqualini R, Arap W, Kleinschmidt JA, Trepel M: Random peptide libraries displayed on adenoassociated virus to select for targeted gene therapy vectors. Nat Biotechnol 2003; 21: 1040-1046.
-
(2003)
Nat Biotechnol
, vol.21
, pp. 1040-1046
-
-
Muller, O.J.1
Kaul, F.2
Weitzman, M.D.3
Pasqualini, R.4
Arap, W.5
Kleinschmidt, J.A.6
Trepel, M.7
-
21
-
-
18444391257
-
Custom adenoassociated virus capsids: The next generation of recombinant vectors with novel tropism
-
Muzyczka N, Warrington KH Jr: Custom adenoassociated virus capsids: the next generation of recombinant vectors with novel tropism. Hum Gene Ther 2005; 16: 408-416.
-
(2005)
Hum Gene Ther
, vol.16
, pp. 408-416
-
-
Muzyczka, N.1
Warrington Jr., K.H.2
-
22
-
-
4444352441
-
Crossdressing the virion: The transcapsidation of adeno-associated virus serotypes functionally defines subgroups
-
Rabinowitz JE, Bowles DE, Faust SM, Ledford JG, Cunningham SE, Samulski RJ: Crossdressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups. J Virol 2004; 78: 4421-4432.
-
(2004)
J Virol
, vol.78
, pp. 4421-4432
-
-
Rabinowitz, J.E.1
Bowles, D.E.2
Faust, S.M.3
Ledford, J.G.4
Cunningham, S.E.5
Samulski, R.J.6
-
23
-
-
38349168183
-
Adeno-associated virus type 12 (AAV12): A novel AAV serotype with sialic acid- and heparan sulfate proteoglycan-independent transduction activity
-
Schmidt M, Voutetakis A, Afione S, Zheng C, Mandikian D, Chiorini JA: Adeno-associated virus type 12 (AAV12): a novel AAV serotype with sialic acid- and heparan sulfate proteoglycan-independent transduction activity. J Virol 2008; 82: 1399-1406.
-
(2008)
J Virol
, vol.82
, pp. 1399-1406
-
-
Schmidt, M.1
Voutetakis, A.2
Afione, S.3
Zheng, C.4
Mandikian, D.5
Chiorini, J.A.6
-
24
-
-
32344434062
-
Bead-based multiplex genotyping of human papillomaviruses
-
Schmitt M, Bravo IG, Snijders PJ, Gissmann L, Pawlita M, Waterboer T: Bead-based multiplex genotyping of human papillomaviruses. J Clin Microbiol 2006; 44: 504-512.
-
(2006)
J Clin Microbiol
, vol.44
, pp. 504-512
-
-
Schmitt, M.1
Bravo, I.G.2
Snijders, P.J.3
Gissmann, L.4
Pawlita, M.5
Waterboer, T.6
-
25
-
-
40749088907
-
Homogeneous amplification of genital human alpha papillomaviruses by PCR using novel broad-spectrum GP5+ and GP6+ primers
-
Schmitt M, Dondog B, Waterboer T, Pawlita M: Homogeneous amplification of genital human alpha papillomaviruses by PCR using novel broad-spectrum GP5+ and GP6+ primers. J Clin Microbiol 2008; 46: 1050-1059.
-
(2008)
J Clin Microbiol
, vol.46
, pp. 1050-1059
-
-
Schmitt, M.1
Dondog, B.2
Waterboer, T.3
Pawlita, M.4
-
26
-
-
70350690548
-
High-throughput detection and multiplex identification of cell contaminations
-
Schmitt M, Pawlita M: High-throughput detection and multiplex identification of cell contaminations. Nucleic Acids Res 2009; 37:e119.
-
(2009)
Nucleic Acids Res
, vol.37
-
-
Schmitt, M.1
Pawlita, M.2
-
27
-
-
0034814413
-
Insertional mutagenesis of the adeno-associated virus type 2 (AAV2) capsid gene and generation of AAV2 vectors targeted to alternative cell-surface receptors
-
Shi W, Arnold GS, Bartlett JS: Insertional mutagenesis of the adeno-associated virus type 2 (AAV2) capsid gene and generation of AAV2 vectors targeted to alternative cell-surface receptors. Hum Gene Ther 2001; 12: 1697-1711.
-
(2001)
Hum Gene Ther
, vol.12
, pp. 1697-1711
-
-
Shi, W.1
Arnold, G.S.2
Bartlett, J.S.3
-
28
-
-
33645456779
-
Insertional mutagenesis at positions 520 and 584 of adenoassociated virus type 2 (AAV2) capsid gene and generation of AAV2 vectors with eliminated heparin-binding ability and introduced novel tropism
-
Shi X, Fang G, Shi W, Bartlett JS: Insertional mutagenesis at positions 520 and 584 of adenoassociated virus type 2 (AAV2) capsid gene and generation of AAV2 vectors with eliminated heparin-binding ability and introduced novel tropism. Hum Gene Ther 2006; 17: 353-361.
-
(2006)
Hum Gene Ther
, vol.17
, pp. 353-361
-
-
Shi, X.1
Fang, G.2
Shi, W.3
Bartlett, J.S.4
-
29
-
-
0027968068
-
ClustalW: Improving the sensitivity of progressive multiple sequence alignment through sequence weighting, position-specific gap penalties and weight matrix choice
-
Thompson JD, Higgins DG, Gibson TJ: ClustalW: improving the sensitivity of progressive multiple sequence alignment through sequence weighting, position-specific gap penalties and weight matrix choice. Nucleic Acids Res 1994; 22: 4673-4680.
-
(1994)
Nucleic Acids Res
, vol.22
, pp. 4673-4680
-
-
Thompson, J.D.1
Higgins, D.G.2
Gibson, T.J.3
-
30
-
-
62549159967
-
Tailoring the AAV vector capsid for gene therapy
-
Vandenberghe LH, Wilson JM, Gao G: Tailoring the AAV vector capsid for gene therapy. Gene Ther 2009; 16: 311-319.
-
(2009)
Gene Ther
, vol.16
, pp. 311-319
-
-
Vandenberghe, L.H.1
Wilson, J.M.2
Gao, G.3
-
31
-
-
67349159102
-
Adeno-associated virus capsid serotype identification: Snalytical methods development and application
-
Van Vliet K, Mohiuddin Y, McClung S, Blouin V, Rolling F, Moullier P, Agbandje-McKenna M, Snyder RO: Adeno-associated virus capsid serotype identification: analytical methods development and application. J Virol Methods 2009; 159: 167-177.
-
(2009)
J Virol Methods
, vol.159
, pp. 167-177
-
-
Van Vliet, K.1
Mohiuddin, Y.2
McClung, S.3
Blouin, V.4
Rolling, F.5
Moullier, P.6
Agbandje-Mckenna, M.7
Snyder, R.O.8
-
32
-
-
0036377851
-
A phase II, double-blind, randomized, placebo-controlled clinical trial of TGAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies
-
Wagner JA, Nepomuceno IB, Messner AH, Moran ML, Batson EP, Dimiceli S, Brown BW, Desch JK, Norbash AM, Conrad CK, Guggino WB, Flotte TR, Wine JJ, Carter BJ, Reynolds TC, Moss RB, Gardner P: A phase II, double-blind, randomized, placebo-controlled clinical trial of TGAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies. Hum Gene Ther 2002; 13: 1349-1359.
-
(2002)
Hum Gene Ther
, vol.13
, pp. 1349-1359
-
-
Wagner, J.A.1
Nepomuceno, I.B.2
Messner, A.H.3
Moran, M.L.4
Batson, E.P.5
Dimiceli, S.6
Brown, B.W.7
Desch, J.K.8
Norbash, A.M.9
Conrad, C.K.10
Guggino, W.B.11
Flotte, T.R.12
Wine, J.J.13
Carter, B.J.14
Reynolds, T.C.15
Moss, R.B.16
Gardner, P.17
-
33
-
-
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, Bellinger DA, Verma IM: Sustained expression of therapeutic level of factor IX in hemophilia b dogs by AAV-mediated gene therapy in liver. Mol Ther 2000; 1: 154-158.
-
(2000)
Mol Ther
, vol.1
, pp. 154-158
-
-
Wang, L.1
Nichols, T.C.2
Read, M.S.3
Bellinger, D.A.4
Verma, I.M.5
-
34
-
-
1342268978
-
Development of efficient viral vectors selective for vascular smooth muscle cells
-
Work LM, Nicklin SA, Brain NJ, Dishart KL, Von Seggern DJ, Hallek M, Buning H, Baker AH: Development of efficient viral vectors selective for vascular smooth muscle cells. Mol Ther 2004; 9: 198-208.
-
(2004)
Mol Ther
, vol.9
, pp. 198-208
-
-
Work, L.M.1
Nicklin, S.A.2
Brain, N.J.3
Dishart, K.L.4
Von Seggern, D.J.5
Hallek, M.6
Buning, H.7
Baker, A.H.8
-
35
-
-
68949202566
-
Transient transfection methods for clinical adeno-associated viral vector production
-
Wright JF: Transient transfection methods for clinical adeno-associated viral vector production. Hum Gene Ther 2009; 20: 698-706.
-
(2009)
Hum Gene Ther
, vol.20
, pp. 698-706
-
-
Wright, J.F.1
-
36
-
-
33746874152
-
Adeno-associated virus serotypes: Vector toolkit for human gene therapy
-
Wu Z, Asokan A, Samulski RJ: Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther 2006; 14: 316-327.
-
(2006)
Mol Ther
, vol.14
, pp. 316-327
-
-
Wu, Z.1
Asokan, A.2
Samulski, R.J.3
|