-
1
-
-
84866899464
-
Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union
-
Ylä-Herttuala, S., Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union. Mol. Ther. 20 (2012), 1831–1832.
-
(2012)
Mol. Ther.
, vol.20
, pp. 1831-1832
-
-
Ylä-Herttuala, S.1
-
2
-
-
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., Cideciyan, A.V., Schwartz, S.B., Wang, L., Conlon, T.J., Boye, S.L., Flotte, T.R., Byrne, B.J., Jacobson, S.G., 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 (2008), 979–990.
-
(2008)
Hum. Gene Ther.
, vol.19
, pp. 979-990
-
-
Hauswirth, W.W.1
Aleman, T.S.2
Kaushal, S.3
Cideciyan, A.V.4
Schwartz, S.B.5
Wang, L.6
Conlon, T.J.7
Boye, S.L.8
Flotte, T.R.9
Byrne, B.J.10
Jacobson, S.G.11
-
3
-
-
44249085878
-
Safety and efficacy of gene transfer for Leber's congenital amaurosis
-
Maguire, A.M., Simonelli, F., Pierce, E.A., Pugh, E.N. Jr., Mingozzi, F., Bennicelli, J., Banfi, S., Marshall, K.A., Testa, F., Surace, E.M., et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N. Engl. J. Med. 358 (2008), 2240–2248.
-
(2008)
N. Engl. J. Med.
, vol.358
, pp. 2240-2248
-
-
Maguire, A.M.1
Simonelli, F.2
Pierce, E.A.3
Pugh, E.N.4
Mingozzi, F.5
Bennicelli, J.6
Banfi, S.7
Marshall, K.A.8
Testa, F.9
Surace, E.M.10
-
4
-
-
70350620424
-
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
-
Maguire, A.M., High, K.A., Auricchio, A., Wright, J.F., Pierce, E.A., Testa, F., Mingozzi, F., Bennicelli, J.L., Ying, G.S., Rossi, S., et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet 374 (2009), 1597–1605.
-
(2009)
Lancet
, vol.374
, pp. 1597-1605
-
-
Maguire, A.M.1
High, K.A.2
Auricchio, A.3
Wright, J.F.4
Pierce, E.A.5
Testa, F.6
Mingozzi, F.7
Bennicelli, J.L.8
Ying, G.S.9
Rossi, S.10
-
5
-
-
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., Pierce, E.A., Mingozzi, F., Bennicelli, J.L., Rossi, S., Marshall, K., Banfi, S., Surace, E.M., et al. Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol. Ther. 18 (2010), 643–650.
-
(2010)
Mol. Ther.
, vol.18
, pp. 643-650
-
-
Simonelli, F.1
Maguire, A.M.2
Testa, F.3
Pierce, E.A.4
Mingozzi, F.5
Bennicelli, J.L.6
Rossi, S.7
Marshall, K.8
Banfi, S.9
Surace, E.M.10
-
6
-
-
84855161388
-
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
-
Nathwani, A.C., Tuddenham, E.G., Rangarajan, S., Rosales, C., McIntosh, J., Linch, D.C., Chowdary, P., Riddell, A., Pie, A.J., Harrington, C., et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N. Engl. J. Med. 365 (2011), 2357–2365.
-
(2011)
N. Engl. J. Med.
, vol.365
, pp. 2357-2365
-
-
Nathwani, A.C.1
Tuddenham, E.G.2
Rangarajan, S.3
Rosales, C.4
McIntosh, J.5
Linch, D.C.6
Chowdary, P.7
Riddell, A.8
Pie, A.J.9
Harrington, C.10
-
7
-
-
84976319493
-
In vivo selection yields AAV-B1 capsid for central nervous system and muscle gene therapy
-
Choudhury, S.R., Fitzpatrick, Z., Harris, A.F., Maitland, S.A., Ferreira, J.S., Zhang, Y., Ma, S., Sharma, R.B., Gray-Edwards, H.L., Johnson, J.A., et al. In vivo selection yields AAV-B1 capsid for central nervous system and muscle gene therapy. Mol. Ther. 24 (2016), 1247–1257.
-
(2016)
Mol. Ther.
, vol.24
, pp. 1247-1257
-
-
Choudhury, S.R.1
Fitzpatrick, Z.2
Harris, A.F.3
Maitland, S.A.4
Ferreira, J.S.5
Zhang, Y.6
Ma, S.7
Sharma, R.B.8
Gray-Edwards, H.L.9
Johnson, J.A.10
-
8
-
-
44949131860
-
In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses
-
Grimm, D., Lee, J.S., Wang, L., Desai, T., Akache, B., Storm, T.A., Kay, M.A., In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. J. Virol. 82 (2008), 5887–5911.
-
(2008)
J. Virol.
, vol.82
, pp. 5887-5911
-
-
Grimm, D.1
Lee, J.S.2
Wang, L.3
Desai, T.4
Akache, B.5
Storm, T.A.6
Kay, M.A.7
-
9
-
-
78651278905
-
Production, purification and characterization of adeno-associated vectors
-
Ayuso, E., Mingozzi, F., Bosch, F., Production, purification and characterization of adeno-associated vectors. Curr. Gene Ther. 10 (2010), 423–436.
-
(2010)
Curr. Gene Ther.
, vol.10
, pp. 423-436
-
-
Ayuso, E.1
Mingozzi, F.2
Bosch, F.3
-
10
-
-
0034328476
-
Scaleable chromatographic purification process for recombinant adeno-associated virus (rAAV)
-
O'Riordan, C.R., Lachapelle, A.L., Vincent, K.A., Wadsworth, S.C., Scaleable chromatographic purification process for recombinant adeno-associated virus (rAAV). J. Gene Med. 2 (2000), 444–454.
-
(2000)
J. Gene Med.
, vol.2
, pp. 444-454
-
-
O'Riordan, C.R.1
Lachapelle, A.L.2
Vincent, K.A.3
Wadsworth, S.C.4
-
11
-
-
79952191532
-
PEG-modulated column chromatography for purification of recombinant adeno-associated virus serotype 9
-
Zhou, J., Yang, X., Wright, J.F., High, K.A., Couto, L., Qu, G., PEG-modulated column chromatography for purification of recombinant adeno-associated virus serotype 9. J. Virol. Methods 173 (2011), 99–107.
-
(2011)
J. Virol. Methods
, vol.173
, pp. 99-107
-
-
Zhou, J.1
Yang, X.2
Wright, J.F.3
High, K.A.4
Couto, L.5
Qu, G.6
-
12
-
-
4644321610
-
Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock
-
Davidoff, A.M., Ng, C.Y., Sleep, S., Gray, J., Azam, S., Zhao, Y., McIntosh, J.H., Karimipoor, M., Nathwani, A.C., Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock. J. Virol. Methods 121 (2004), 209–215.
-
(2004)
J. Virol. Methods
, vol.121
, pp. 209-215
-
-
Davidoff, A.M.1
Ng, C.Y.2
Sleep, S.3
Gray, J.4
Azam, S.5
Zhao, Y.6
McIntosh, J.H.7
Karimipoor, M.8
Nathwani, A.C.9
-
13
-
-
84896368264
-
OneBac: platform for scalable and high-titer production of adeno-associated virus serotype 1-12 vectors for gene therapy
-
Mietzsch, M., Grasse, S., Zurawski, C., Weger, S., Bennett, A., Agbandje-McKenna, M., Muzyczka, N., Zolotukhin, S., Heilbronn, R., OneBac: platform for scalable and high-titer production of adeno-associated virus serotype 1-12 vectors for gene therapy. Hum. Gene Ther. 25 (2014), 212–222.
-
(2014)
Hum. Gene Ther.
, vol.25
, pp. 212-222
-
-
Mietzsch, M.1
Grasse, S.2
Zurawski, C.3
Weger, S.4
Bennett, A.5
Agbandje-McKenna, M.6
Muzyczka, N.7
Zolotukhin, S.8
Heilbronn, R.9
-
14
-
-
33646919907
-
Isolation of an adeno-associated virus (AAV)-specific camelid-derived single chain antibody fragment: a novel tool for purification of AAV vectors of different serotypes
-
Oranje, P.P.A., Verheesen, P., Verbart, D., Mijnsbergen, Y., Haard, H.J.W., Hermans, P., et al. Isolation of an adeno-associated virus (AAV)-specific camelid-derived single chain antibody fragment: a novel tool for purification of AAV vectors of different serotypes. Mol. Ther., 9, 2004, S162.
-
(2004)
Mol. Ther.
, vol.9
, pp. S162
-
-
Oranje, P.P.A.1
Verheesen, P.2
Verbart, D.3
Mijnsbergen, Y.4
Haard, H.J.W.5
Hermans, P.6
-
15
-
-
85015250032
-
Identification of an adeno-associated virus binding epitope for AVB sepharose affinity resin
-
Wang, Q., Lock, M., Prongay, A.J., Alvira, M.R., Petkov, B., Wilson, J.M., Identification of an adeno-associated virus binding epitope for AVB sepharose affinity resin. Mol. Ther. Methods Clin. Dev., 2, 2015, 15040.
-
(2015)
Mol. Ther. Methods Clin. Dev.
, vol.2
, pp. 15040
-
-
Wang, Q.1
Lock, M.2
Prongay, A.J.3
Alvira, M.R.4
Petkov, B.5
Wilson, J.M.6
-
16
-
-
33644820684
-
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
-
Manno, C.S., Pierce, G.F., Arruda, V.R., Glader, B., Ragni, M., Rasko, J.J., Ozelo, M.C., Hoots, K., Blatt, P., Konkle, B., et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat. Med. 12 (2006), 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
-
17
-
-
33846636272
-
Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography
-
Qu, G., Bahr-Davidson, J., Prado, J., Tai, A., Cataniag, F., McDonnell, J., Zhou, J., Hauck, B., Luna, J., Sommer, J.M., et al. Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography. J. Virol. Methods 140 (2007), 183–192.
-
(2007)
J. Virol. Methods
, vol.140
, 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
-
18
-
-
84949634261
-
Analytical ultracentrifugation as an approach to characterize recombinant adeno-associated viral vectors
-
Burnham, B., Nass, S., Kong, E., Mattingly, M., Woodcock, D., Song, A., Wadsworth, S., Cheng, S.H., Scaria, A., O'Riordan, C.R., Analytical ultracentrifugation as an approach to characterize recombinant adeno-associated viral vectors. Hum. Gene Ther. Methods 26 (2015), 228–242.
-
(2015)
Hum. Gene Ther. Methods
, vol.26
, pp. 228-242
-
-
Burnham, B.1
Nass, S.2
Kong, E.3
Mattingly, M.4
Woodcock, D.5
Song, A.6
Wadsworth, S.7
Cheng, S.H.8
Scaria, A.9
O'Riordan, C.R.10
-
19
-
-
35148850085
-
AAV-mediated expression targeting of rod and cone photoreceptors with a human rhodopsin kinase promoter
-
Khani, S.C., Pawlyk, B.S., Bulgakov, O.V., Kasperek, E., Young, J.E., Adamian, M., Sun, X., Smith, A.J., Ali, R.R., Li, T., AAV-mediated expression targeting of rod and cone photoreceptors with a human rhodopsin kinase promoter. Invest. Ophthalmol. Vis. Sci. 48 (2007), 3954–3961.
-
(2007)
Invest. Ophthalmol. Vis. Sci.
, vol.48
, pp. 3954-3961
-
-
Khani, S.C.1
Pawlyk, B.S.2
Bulgakov, O.V.3
Kasperek, E.4
Young, J.E.5
Adamian, M.6
Sun, X.7
Smith, A.J.8
Ali, R.R.9
Li, T.10
-
20
-
-
84983132701
-
Structure of neurotropic adeno-associated virus AAVrh.8
-
Halder, S., Van Vliet, K., Smith, J.K., Duong, T.T., McKenna, R., Wilson, J.M., Agbandje-McKenna, M., Structure of neurotropic adeno-associated virus AAVrh.8. J. Struct. Biol. 192 (2015), 21–36.
-
(2015)
J. Struct. Biol.
, vol.192
, pp. 21-36
-
-
Halder, S.1
Van Vliet, K.2
Smith, J.K.3
Duong, T.T.4
McKenna, R.5
Wilson, J.M.6
Agbandje-McKenna, M.7
-
21
-
-
43949105839
-
Surface loop dynamics in adeno-associated virus capsid assembly
-
DiPrimio, N., Asokan, A., Govindasamy, L., Agbandje-McKenna, M., Samulski, R.J., Surface loop dynamics in adeno-associated virus capsid assembly. J. Virol. 82 (2008), 5178–5189.
-
(2008)
J. Virol.
, vol.82
, pp. 5178-5189
-
-
DiPrimio, N.1
Asokan, A.2
Govindasamy, L.3
Agbandje-McKenna, M.4
Samulski, R.J.5
-
22
-
-
0035875669
-
DNA helicase-mediated packaging of adeno-associated virus type 2 genomes into preformed capsids
-
King, J.A., Dubielzig, R., Grimm, D., Kleinschmidt, J.A., DNA helicase-mediated packaging of adeno-associated virus type 2 genomes into preformed capsids. EMBO J. 20 (2001), 3282–3291.
-
(2001)
EMBO J.
, vol.20
, pp. 3282-3291
-
-
King, J.A.1
Dubielzig, R.2
Grimm, D.3
Kleinschmidt, J.A.4
-
23
-
-
0033022953
-
Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield
-
Zolotukhin, S., Byrne, B.J., Mason, E., Zolotukhin, I., Potter, M., Chesnut, K., Summerford, C., Samulski, R.J., Muzyczka, N., Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther. 6 (1999), 973–985.
-
(1999)
Gene Ther.
, vol.6
, pp. 973-985
-
-
Zolotukhin, S.1
Byrne, B.J.2
Mason, E.3
Zolotukhin, I.4
Potter, M.5
Chesnut, K.6
Summerford, C.7
Samulski, R.J.8
Muzyczka, N.9
-
24
-
-
2642642141
-
Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus
-
Xiao, X., Li, J., Samulski, R.J., Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J. Virol. 72 (1998), 2224–2232.
-
(1998)
J. Virol.
, vol.72
, pp. 2224-2232
-
-
Xiao, X.1
Li, J.2
Samulski, R.J.3
-
25
-
-
74949114697
-
Manufacturing recombinant adeno-associated viral vectors from producer cell clones
-
Thorne, B.A., Takeya, R.K., Peluso, R.W., Manufacturing recombinant adeno-associated viral vectors from producer cell clones. Hum. Gene Ther. 20 (2009), 707–714.
-
(2009)
Hum. Gene Ther.
, vol.20
, pp. 707-714
-
-
Thorne, B.A.1
Takeya, R.K.2
Peluso, R.W.3
-
26
-
-
84883020987
-
Generation and characterization of adeno-associated virus producer cell lines for research and preclinical vector production
-
Martin, J., Frederick, A., Luo, Y., Jackson, R., Joubert, M., Sol, B., Poulin, F., Pastor, E., Armentano, D., Wadsworth, S., Vincent, K., Generation and characterization of adeno-associated virus producer cell lines for research and preclinical vector production. Hum. Gene Ther. Methods 24 (2013), 253–269.
-
(2013)
Hum. Gene Ther. Methods
, vol.24
, pp. 253-269
-
-
Martin, J.1
Frederick, A.2
Luo, Y.3
Jackson, R.4
Joubert, M.5
Sol, B.6
Poulin, F.7
Pastor, E.8
Armentano, D.9
Wadsworth, S.10
Vincent, K.11
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