-
1
-
-
79955405275
-
Good manufacturing practice production of self-complementary serotype 8 adenoassociated viral vector for a hemophilia B clinical trial
-
Allay, J.A., Sleep, S., Long, S., et al. (2011). Good manufacturing practice production of self-complementary serotype 8 adenoassociated viral vector for a hemophilia B clinical trial. Hum. Gene Ther. 22, 595-604.
-
(2011)
Hum. Gene Ther
, vol.22
, pp. 595-604
-
-
Allay, J.A.1
Sleep, S.2
Long, S.3
-
2
-
-
77950858020
-
High AAV vector purity results in serotype-and tissue-independent enhancement of transduction efficiency
-
Ayuso, E., Mingozzi, F., Montane, J., et al. (2010). High AAV vector purity results in serotype-and tissue-independent enhancement of transduction efficiency. Gene Ther. 17, 503-510.
-
(2010)
Gene Ther
, vol.17
, pp. 503-510
-
-
Ayuso, E.1
Mingozzi, F.2
Montane, J.3
-
3
-
-
44249120315
-
Effect of gene therapy on visual function in Leber's congenital amaurosis
-
DOI 10.1056/NEJMoa0802268
-
Bainbridge, J.W., Smith, A.J., Barker, S.S., et al. (2008). Effect of gene therapy on visual function in Leber's congenital amaurosis. N. Engl. J. Med. 358, 2231-2239. (Pubitemid 351724452)
-
(2008)
New England Journal of Medicine
, vol.358
, Issue.21
, pp. 2231-2239
-
-
Bainbridge, J.W.B.1
Smith, A.J.2
Barker, S.S.3
Robbie, S.4
Henderson, R.5
Balaggan, K.6
Viswanathan, A.7
Holder, G.E.8
Stockman, A.9
Tyler, N.10
Petersen-Jones, S.11
Bhattacharya, S.S.12
Thrasher, A.J.13
Fitzke, F.W.14
Carter, B.J.15
Rubin, G.S.16
Moore, A.T.17
Ali, R.R.18
-
4
-
-
0036852208
-
A versatile and scalable two-step ion-exchange chromatography process for the purification of recombinant adeno-associated virus serotypes-2 and -5
-
DOI 10.1016/S1525-0016(02)90719-7
-
Brument, N., Morenweiser, R., Blouin, V., et al. (2002). A versatile and scalable two-step ion-exchange chromatography process for the purification of recombinant adeno-associated virus serotypes 2 and 5. Mol. Ther. 6, 678-686. (Pubitemid 35363701)
-
(2002)
Molecular Therapy
, vol.6
, Issue.5
, pp. 678-686
-
-
Brument, N.1
Morenweiser, R.2
Blouin, V.3
Toublanc, E.4
Raimbaud, I.5
Cherel, Y.6
Folliot, S.7
Gaden, F.8
Boulanger, P.9
Kroner-Lux, G.10
Moullier, P.11
Rolling, F.12
Salvetti, A.13
-
5
-
-
25144482280
-
Evidence for encapsidation of prokaryotic sequences during recombinant adeno-associated virus production and their in vivo persistence after vector delivery
-
DOI 10.1016/j.ymthe.2005.06.003, PII S1525001605002303
-
Chadeuf, G., Ciron, C., Moullier, P., and Salvetti, A. (2005). Evidence for encapsidation of prokaryotic sequences during recombinant adeno-associated virus production and their in vivo persistence after vector delivery. Mol. Ther. 12, 744-753. (Pubitemid 41350156)
-
(2005)
Molecular Therapy
, vol.12
, Issue.4
, pp. 744-753
-
-
Chadeuf, G.1
Ciron, C.2
Moullier, P.3
Salvetti, A.4
-
6
-
-
73449128979
-
Safety and tolerability of putaminal AADC gene therapy for Parkinson disease
-
Christine, C.W., Starr, P.A., Larson, P.S., 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
-
7
-
-
68949210394
-
Large-scale adeno-associated viral vector production using a herpesvirusbased system enables manufacturing for clinical studies
-
Clement, N., Knop, D.R., and Byrne, B.J. (2009). Large-scale adeno-associated viral vector production using a herpesvirusbased system enables manufacturing for clinical studies. Hum. Gene Ther. 20, 796-806.
-
(2009)
Hum. Gene Ther
, vol.20
, pp. 796-806
-
-
Clement, N.1
Knop, D.R.2
Byrne, B.J.3
-
8
-
-
4644321610
-
Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock
-
DOI 10.1016/j.jviromet.2004.07.001, PII S0166093404001922
-
Davidoff, A.M., Ng, C.Y., Sleep, S., et al. (2004). Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock. J. Virol. Methods 121, 209-215. (Pubitemid 39295711)
-
(2004)
Journal of Virological Methods
, vol.121
, Issue.2
, pp. 209-215
-
-
Davidoff, A.M.1
Ng, C.Y.C.2
Sleep, S.3
Gray, J.4
Azam, S.5
Zhao, Y.6
McIntosh, J.H.7
Karimipoor, M.8
Nathwani, A.C.9
-
9
-
-
44949222522
-
Results from a phase i safety trial of hAADC gene therapy for Parkinson disease
-
Eberling, J.L., Jagust, W.J., Christine, C.W., 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
-
10
-
-
0032773889
-
Titration of AAV-2 particles via a novel capsid ELISA: Packaging of genomes can limit production of recombinant AAV-2
-
DOI 10.1038/sj.gt.3300946
-
Grimm, D., Kern, A., Pawlita, M., et al. (1999). Titration of AAV-2 particles via a novel capsid ELISA: Packaging of genomes can limit production of recombinant AAV-2. Gene Ther. 6, 1322-1330. (Pubitemid 29359028)
-
(1999)
Gene Therapy
, vol.6
, Issue.7
, pp. 1322-1330
-
-
Grimm, D.1
Kern, A.2
Pawlita, M.3
Ferrari, F.K.4
Samulski, R.J.5
Kleinschmidt, J.A.6
-
11
-
-
79953793932
-
Capsid-expressing DNA in AAV vectors and its elimination by use of an oversize capsid gene for vector production
-
Halbert, C.L., Metzger, M.J., Lam, S.L., and Miller, A.D. (2011). Capsid-expressing DNA in AAV vectors and its elimination by use of an oversize capsid gene for vector production. Gene Ther. 18, 411-417.
-
(2011)
Gene Ther
, vol.18
, pp. 411-417
-
-
Halbert, C.L.1
Metzger, M.J.2
Lam, S.L.3
Miller, A.D.4
-
12
-
-
54949104686
-
Treatment of Leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: Short-term results of a phase i trial
-
Hauswirth, W.W., Aleman, T.S., Kaushal, S., et al. (2008). Treatment of Leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: Short-term results of a phase I trial. Hum. Gene Ther. 19, 979-990.
-
(2008)
Hum. Gene Ther
, vol.19
, pp. 979-990
-
-
Hauswirth, W.W.1
Aleman, T.S.2
Kaushal, S.3
-
13
-
-
0036019204
-
Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography
-
DOI 10.1089/104303402320139014
-
Kaludov, N., Handelman, B., and Chiorini, J.A. (2002). Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography. Hum. Gene Ther. 13, 1235-1243. (Pubitemid 34746703)
-
(2002)
Human Gene Therapy
, vol.13
, Issue.10
, pp. 1235-1243
-
-
Kaludov, N.1
Handelman, B.2
Chiorini, J.A.3
-
14
-
-
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
-
DOI 10.1016/S0140-6736(07)60982-9, PII S0140673607609829
-
Kaplitt, M.G., Feigin, A., Tang, C., et al. (2007). 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 369, 2097-2105. (Pubitemid 46935946)
-
(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
-
15
-
-
77958104835
-
Rapid, simple, and versatile manufacturing of recombinant adenoassociated viral vectors at scale
-
Lock, M., Alvira, M., Vandenberghe, L.H., et al. (2010a). Rapid, simple, and versatile manufacturing of recombinant adenoassociated viral vectors at scale. Hum. Gene Ther. 21, 1259-1271.
-
(2010)
Hum. Gene Ther
, vol.21
, pp. 1259-1271
-
-
Lock, M.1
Alvira, M.2
Vandenberghe, L.H.3
-
16
-
-
77958099436
-
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material
-
Lock, M., McGorray, S., Auricchio, A., et al. (2010b). Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Hum. Gene Ther. 21, 1273-1285.
-
(2010)
Hum. Gene Ther
, vol.21
, pp. 1273-1285
-
-
Lock, M.1
McGorray, S.2
Auricchio, A.3
-
17
-
-
44249085878
-
Safety and efficacy of gene transfer for Leber's congenital amaurosis
-
DOI 10.1056/NEJMoa0802315
-
Maguire, A.M., Simonelli, F., Pierce, E.A., et al. (2008). Safety and efficacy of gene transfer for Leber's congenital amaurosis. N. Engl. J. Med. 358, 2240-2248. (Pubitemid 351724453)
-
(2008)
New England Journal of Medicine
, vol.358
, Issue.21
, pp. 2240-2248
-
-
Maguire, A.M.1
Simonelli, F.2
Pierce, E.A.3
Pugh Jr., E.N.4
Mingozzi, F.5
Bennicelli, J.6
Banfi, S.7
Marshall, K.A.8
Testa, F.9
Surace, E.M.10
Rossi, S.11
Lyubarsky, A.12
Arruda, V.R.13
Konkle, B.14
Stone, E.15
Sun, J.16
Jacobs, J.17
Dell'Osso, L.18
Hertle, R.19
Ma, J.-X.20
Redmond, T.M.21
Zhu, X.22
Hauck, B.23
Zelenaia, O.24
Shindler, K.S.25
Maguire, M.G.26
Wright, J.F.27
Volpe, N.J.28
McDonnell, J.W.29
Auricchio, A.30
High, K.A.31
Bennett, J.32
more..
-
18
-
-
70350620424
-
Agedependent effects of RPE65 gene therapy for Leber's congenital amaurosis: A phase 1 dose-escalation trial
-
Maguire, A.M., High, K.A., Auricchio, A., et al. (2009). Agedependent effects of RPE65 gene therapy for Leber's congenital amaurosis: A phase 1 dose-escalation trial. Lancet 374, 1597-1605.
-
(2009)
Lancet
, vol.374
, pp. 1597-1605
-
-
Maguire, A.M.1
High, K.A.2
Auricchio, A.3
-
19
-
-
34147098413
-
+ T-cell responses to adeno-associated virus capsid in humans
-
DOI 10.1038/nm1549, PII NM1549
-
Mingozzi, F., Maus, M.V., Hui, D.J., et al. (2007). CD8+ T-cell responses to adeno-associated virus capsid in humans. Nat. Med. 13, 419-422. (Pubitemid 46559826)
-
(2007)
Nature Medicine
, vol.13
, Issue.4
, pp. 419-422
-
-
Mingozzi, F.1
Maus, M.V.2
Hui, D.J.3
Sabatino, D.E.4
Murphy, S.L.5
Rasko, J.E.J.6
Ragni, M.V.7
Manno, C.S.8
Sommer, J.9
Jiang, H.10
Pierce, G.F.11
Ertl, H.C.J.12
High, K.A.13
-
20
-
-
45749101234
-
International efforts for recombinant adeno-associated viral vector reference standards
-
DOI 10.1038/mt.2008.125, PII MT2008125
-
Moullier, P., and Snyder, R.O. (2008). International efforts for recombinant adeno-associated viral vector reference standards. Mol. Ther. 16, 1185-1188. (Pubitemid 351866048)
-
(2008)
Molecular Therapy
, vol.16
, Issue.7
, pp. 1185-1188
-
-
Moullier, P.1
Snyder, R.O.2
-
21
-
-
70349129594
-
Scalable purification of adeno-associated virus serotype 1 (AAV1) and AAV8 vectors, using dual ion-exchange adsorptive membranes
-
Okada, T., Nonaka-Sarukawa, M., Uchibori, R., et al. (2009). Scalable purification of adeno-associated virus serotype 1 (AAV1) and AAV8 vectors, using dual ion-exchange adsorptive membranes. Hum. Gene Ther. 20, 1013-1021.
-
(2009)
Hum. Gene Ther
, vol.20
, pp. 1013-1021
-
-
Okada, T.1
Nonaka-Sarukawa, M.2
Uchibori, R.3
-
22
-
-
33846636272
-
Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography
-
DOI 10.1016/j.jviromet.2006.11.019, PII S0166093406004022
-
Qu, G., Bahr-Davidson, J., Prado, J., et al. (2007). Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography. J. Virol. Methods 140, 183-192. (Pubitemid 46185608)
-
(2007)
Journal of Virological Methods
, vol.140
, Issue.1-2
, pp. 183-192
-
-
Qu, G.1
Bahr-Davidson, J.2
Prado, J.3
Tai, A.4
Cataniag, F.5
McDonnell, J.6
Zhou, J.7
Hauck, B.8
Luna, J.9
Sommer, J.M.10
Smith, P.11
Zhou, S.12
Colosi, P.13
High, K.A.14
Pierce, G.F.15
Wright, J.F.16
-
23
-
-
77649242176
-
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration
-
Simonelli, F., Maguire, A.M., Testa, F., et al. (2010). Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol. Ther. 18, 643-650.
-
(2010)
Mol. Ther
, vol.18
, pp. 643-650
-
-
Simonelli, F.1
Maguire, A.M.2
Testa, F.3
-
24
-
-
70449103126
-
A simplified baculovirus-AAV expression vector system coupled with onestep affinity purification yields high-titer rAAV stocks from insect cells
-
Smith, R.H., Levy, J.R., and Kotin, R.M. (2009). A simplified baculovirus-AAV expression vector system coupled with onestep affinity purification yields high-titer rAAV stocks from insect cells. Mol. Ther. 17, 1888-1896.
-
(2009)
Mol. Ther
, vol.17
, pp. 1888-1896
-
-
Smith, R.H.1
Levy, J.R.2
Kotin, R.M.3
-
25
-
-
0037223085
-
Quantification of adeno-associated virus particles and empty capsids by optical density measurement
-
DOI 10.1016/S1525-0016(02)00019-9
-
Sommer, J.M., Smith, P.H., Parthasarathy, S., et al. (2003). Quantification of adeno-associated virus particles and empty capsids by optical density measurement. Mol. Ther. 7, 122-128. (Pubitemid 36305364)
-
(2003)
Molecular Therapy
, vol.7
, Issue.1
, pp. 122-128
-
-
Sommer, J.M.1
Smith, P.H.2
Parthasarathy, S.3
Isaacs, J.4
Vijay, S.5
Kieran, J.6
Powell, S.K.7
McClelland, A.8
Wright, F.9
-
26
-
-
74949114697
-
Manufacturing recombinant adeno-associated viral vectors from producer cell clones
-
Thorne, B.A., Takeya, R.K., and Peluso, R.W. (2009). Manufacturing recombinant adeno-associated viral vectors from producer cell clones. Hum. Gene Ther. 20, 707-714.
-
(2009)
Hum. Gene Ther
, vol.20
, pp. 707-714
-
-
Thorne, B.A.1
Takeya, R.K.2
Peluso, R.W.3
-
27
-
-
21244503122
-
Mechanisms of adeno-associated virus genome encapsidation
-
DOI 10.2174/1566523054065011
-
Timpe, J., Bevington, J., Casper, J., et al. (2005). Mechanisms of adeno-associated virus genome encapsidation. Curr. Gene Ther. 5, 273-284. (Pubitemid 40895012)
-
(2005)
Current Gene Therapy
, vol.5
, Issue.3
, pp. 273-284
-
-
Timpe, J.1
Bevington, J.2
Casper, J.3
Dignam, J.D.4
Trempe, J.P.5
-
28
-
-
33645516194
-
Removal of empty capsids from type 1 adeno-associated virus vector stocks by anion-exchange chromatography potentiates transgene expression
-
Urabe, M., Xin, K.Q., Obara, Y., et al. (2006). Removal of empty capsids from type 1 adeno-associated virus vector stocks by anion-exchange chromatography potentiates transgene expression. Mol. Ther. 13, 823-828.
-
(2006)
Mol. Ther
, vol.13
, pp. 823-828
-
-
Urabe, M.1
Xin, K.Q.2
Obara, Y.3
-
29
-
-
68949220858
-
Producing recombinant adeno-associated virus in foster cells: Overcoming production limitations using a baculovirus-insect cell expression strategy
-
Virag, T., Cecchini, S., and Kotin, R.M. (2009). Producing recombinant adeno-associated virus in foster cells: Overcoming production limitations using a baculovirus-insect cell expression strategy. Hum. Gene Ther. 20, 807-817.
-
(2009)
Hum. Gene Ther
, vol.20
, pp. 807-817
-
-
Virag, T.1
Cecchini, S.2
Kotin, R.M.3
-
30
-
-
80051993162
-
Adeno-associated viral vector manufacturing: Keeping pace with accelerating clinical development
-
Wright, J.F. (2011a). Adeno-associated viral vector manufacturing: Keeping pace with accelerating clinical development. Hum. Gene Ther. 22, 913-914.
-
(2011)
Hum. Gene Ther
, vol.22
, pp. 913-914
-
-
Wright, J.F.1
-
31
-
-
79955416671
-
New adeno-associated virus strategies to support momentum in the clinic
-
Wright, J.F. (2011b). New adeno-associated virus strategies to support momentum in the clinic. Hum. Gene Ther. 22, 519-521.
-
(2011)
Hum. Gene Ther
, vol.22
, pp. 519-521
-
-
Wright, J.F.1
-
32
-
-
0032924185
-
Gene therapy vectors based on adeno-associated virus type 1
-
Xiao, W., Chirmule, N., Berta, S.C., et al. (1999). Gene therapy vectors based on adeno-associated virus type 1. J. Virol. 73, 3994-4003. (Pubitemid 29189844)
-
(1999)
Journal of Virology
, vol.73
, Issue.5
, pp. 3994-4003
-
-
Xiao, W.1
Chirmule, N.2
Berta, S.C.3
McCullough, B.4
Gao, G.5
Wilson, J.M.6
-
33
-
-
70249151690
-
Adenovirus-adenoassociated virus hybrid for large-scale recombinant adeno-associated virus production
-
Zhang, H., Xie, J., Xie, Q., et al. (2009). Adenovirus-adenoassociated virus hybrid for large-scale recombinant adeno-associated virus production. Hum. Gene Ther. 20, 922-929.
-
(2009)
Hum. Gene Ther
, vol.20
, pp. 922-929
-
-
Zhang, H.1
Xie, J.2
Xie, Q.3
-
34
-
-
79952191532
-
PEG-modulated column chromatography for purification of recombinant adeno-associated virus serotype 9
-
Zhou, J., Yang, X., Wright, J.F., et al. (2011). PEG-modulated column chromatography for purification of recombinant adeno-associated virus serotype 9. J. Virol. Methods 173, 99-107.
-
(2011)
J. Virol. Methods
, vol.173
, pp. 99-107
-
-
Zhou, J.1
Yang, X.2
Wright, J.F.3
|