-
1
-
-
57749169283
-
Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat
-
Bish, L.T., Morine, K., Sleeper, M.M., Sanmiguel, J., Wu, D., Gao, G., Wilson, J.M., and Sweeney, H.L. (2008). Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat. Hum. Gene Ther. 19, 1359-1368.
-
(2008)
Hum. Gene Ther.
, vol.19
, pp. 1359-1368
-
-
Bish, L.T.1
Morine, K.2
Sleeper, M.M.3
Sanmiguel, J.4
Wu, D.5
Gao, G.6
Wilson, J.M.7
Sweeney, H.L.8
-
2
-
-
4644252083
-
Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6
-
Blankinship, M.J., Gregorevic, P., Allen, J.M., Harper, S.Q., Harper, H., Halbert, C.L., Miller, D.A., and Chamberlain, J.S. (2004). Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. Mol. Ther. 10, 671-678.
-
(2004)
Mol. Ther.
, vol.10
, pp. 671-678
-
-
Blankinship, M.J.1
Gregorevic, P.2
Allen, J.M.3
Harper, S.Q.4
Harper, H.5
Halbert, C.L.6
Miller, D.A.7
Chamberlain, J.S.8
-
3
-
-
54249126862
-
Gene therapy using adeno-associated virus vectors
-
Daya, S., and Berns, K.I. (2008). Gene therapy using adeno-associated virus vectors. Clin. Microbiol. Rev. 21, 583-593.
-
(2008)
Clin. Microbiol. Rev.
, vol.21
, pp. 583-593
-
-
Daya, S.1
Berns, K.I.2
-
4
-
-
0029923638
-
Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors
-
Ferrari, F.K., Samulski, T., Shenk, T., and Samulski, R.J. (1996). Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J. Virol. 70, 3227-3234.
-
(1996)
J. Virol.
, vol.70
, pp. 3227-3234
-
-
Ferrari, F.K.1
Samulski, T.2
Shenk, T.3
Samulski, R.J.4
-
5
-
-
21244460769
-
New recombinant serotypes of AAV vectors
-
Gao, G., Vandenberghe, L.H., and Wilson, J.M. (2005). New recombinant serotypes of AAV vectors. Curr. Gene Ther. 5, 285-297.
-
(2005)
Curr. Gene Ther.
, vol.5
, pp. 285-297
-
-
Gao, G.1
Vandenberghe, L.H.2
Wilson, J.M.3
-
6
-
-
0037015049
-
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
-
Gao, G.P., Alvira, M.R., Wang, L., Calcedo, R., Johnston, J., and Wilson, J.M. (2002). Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. U.S.A. 99, 11854-11859.
-
(2002)
Proc. Natl. Acad. Sci. U.S.A.
, 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
-
-
33947247444
-
Efficient whole-body transduction with trans-splicing adeno-associated viral vectors
-
Ghosh, A., Yue, Y., Long, C., Bostick, B., and Duan, D. (2007). Efficient whole-body transduction with trans-splicing adeno-associated viral vectors. Mol. Ther. 15, 750-755.
-
(2007)
Mol. Ther.
, vol.15
, pp. 750-755
-
-
Ghosh, A.1
Yue, Y.2
Long, C.3
Bostick, B.4
Duan, D.5
-
8
-
-
0036127393
-
Modular flexibility of dystrophin: Implications for gene therapy of Du-chenne muscular dystrophy
-
Harper, S.Q., Hauser, M.A., Dellorusso, C., Duan, D., Crawford, R.W., Phelps, S.F., Harper, H.A., Robinson, A.S., Engelhardt, J.F., Brooks, S.V., and Chamberlain, J.S. (2002). Modular flexibility of dystrophin: Implications for gene therapy of Du-chenne muscular dystrophy. Nat. Med. 8, 253-261.
-
(2002)
Nat. Med.
, vol.8
, pp. 253-261
-
-
Harper, S.Q.1
Hauser, M.A.2
Dellorusso, C.3
Duan, D.4
Crawford, R.W.5
Phelps, S.F.6
Harper, H.A.7
Robinson, A.S.8
Engelhardt, J.F.9
Brooks, S.V.10
Chamberlain, J.S.11
-
9
-
-
2342457825
-
Clinical gene transfer studies for hemophilia B
-
High, K.A. (2004). Clinical gene transfer studies for hemophilia B. Semin. Thromb. Hemost. 30, 257-267.
-
(2004)
Semin. Thromb. Hemost.
, vol.30
, pp. 257-267
-
-
High, K.A.1
-
10
-
-
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., and During, M.J. (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.
-
(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
-
11
-
-
0141613937
-
Efficient and long-term intracardiac gene transfer in 5-sarcoglycan-deficiency hamster by adeno-associated virus-2 vectors
-
Li, J., Wang, D., Qian, S., Chen, Z., Zhu, T., and Xiao, X. (2003). Efficient and long-term intracardiac gene transfer in 5-sarcoglycan-deficiency hamster by adeno-associated virus-2 vectors. Gene Ther. 10, 1807-1813
-
(2003)
Gene Ther.
, vol.10
, pp. 1807-1813
-
-
Li, J.1
Wang, D.2
Qian, S.3
Chen, Z.4
Zhu, T.5
Xiao, X.6
-
12
-
-
44249085878
-
Safety and efficacy of gene transfer for Leber's congenital amaurosis
-
Maguire, A.M., Simonelli, F., Pierce, E.A., Pugh, E.N., Jr., Min-gozzi, F., Bennicelli, J., Banfi, S., Marshall, K.A., Testa, F., Surace, E.M., Rossi, S., Lyubarsky, A., Arruda, V.R., Konkle, B., Stone, E., Sun, J., Jacobs, J., Dell'osso, L., Hertle, R., Ma, J.X., Redmond, T.M., Zhu, X., Hauck, B., Zelenaia, O., Shindler, K.S., Maguire, M.G., Wright, J.F., Volpe, N.J., McDonnell, J.W., Auricchio, A., High, K.A., and Bennett, J. (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
Pugh Jr., E.N.4
Min-Gozzi, 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..
-
13
-
-
70350620424
-
Age-dependent effects of RPE65 gene therapy for Le-ber'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., Fulton, A., Marshall, K.A., Banfi, S., Chung, D.C., Morgan, J.I., Hauck, B., Zelenaia, O., Zhu, X., Raffini, L., Coppieters, F., De Baere, E., Shindler, K.S., Volpe, N.J., Surace, E.M., Acerra, C., Lyubarsky, A., Redmond, T.M., Stone, E., Sun, J., McDonnell, J.W., Leroy, B.P., Simonelli, F., and Bennett, J. (2009). Age-dependent effects of RPE65 gene therapy for Le-ber'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
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
Fulton, A.11
Marshall, K.A.12
Banfi, S.13
Chung, D.C.14
Morgan, J.I.15
Hauck, B.16
Zelenaia, O.17
Zhu, X.18
Raffini, L.19
Coppieters, F.20
De Baere, E.21
Shindler, K.S.22
Volpe, N.J.23
Surace, E.M.24
Acerra, C.25
Lyubarsky, A.26
Redmond, T.M.27
Stone, E.28
Sun, J.29
McDonnell, J.W.30
Leroy, B.P.31
Simonelli, F.32
Bennett, J.33
more..
-
14
-
-
0013442744
-
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
-
Manno, C.S., Chew, A.J., Hutchison, S., Larson, P.J., Herzog, R.W., Arruda, V.R., Tai, S.J., Ragni, M.V., Thompson, A., Ozelo, M., Couto, L.B., Leonard, D.G., Johnson, F.A., McClelland, A., Scallan, C., Skarsgard, E., Flake, A.W., Kay, M.A., High, K.A., and Glader, B. (2003). AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101, 2963-2972.
-
(2003)
Blood
, vol.101
, pp. 2963-2972
-
-
Manno, C.S.1
Chew, A.J.2
Hutchison, S.3
Larson, P.J.4
Herzog, R.W.5
Arruda, V.R.6
Tai, S.J.7
Ragni, M.V.8
Thompson, A.9
Ozelo, M.10
Couto, L.B.11
Leonard, D.G.12
Johnson, F.A.13
McClelland, A.14
Scallan, C.15
Skarsgard, E.16
Flake, A.W.17
Kay, M.A.18
High, K.A.19
Glader, B.20
more..
-
15
-
-
18444391257
-
Custom ade-no-associated virus capsids: The next generation of re-combinant vectors with novel tropism
-
Muzyczka, N., and Warrington, K.H., Jr. (2005). Custom ade-no-associated virus capsids: The next generation of re-combinant vectors with novel tropism. Hum. Gene Ther. 16, 408-416.
-
(2005)
Hum. Gene Ther.
, vol.16
, pp. 408-416
-
-
Muzyczka, N.1
Warrington Jr., K.H.2
-
16
-
-
0010390169
-
Cloning of adeno-associated virus into pBR322: Rescue of in-tact virus from the recombinant plasmid in human cells
-
Samulski, R.J., Berns, K.I., Tan, M., and Muzyczka, N. (1982). Cloning of adeno-associated virus into pBR322: Rescue of in-tact virus from the recombinant plasmid in human cells. Proc. Natl. Acad. Sci. U.S.A. 79, 2077-2081.
-
(1982)
Proc. Natl. Acad. Sci. U.S.A.
, vol.79
, pp. 2077-2081
-
-
Samulski, R.J.1
Berns, K.I.2
Tan, M.3
Muzyczka, N.4
-
17
-
-
0034610364
-
Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
-
Wang, B., Li, J., and Xiao, X. (2000). Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. Proc. Natl. Acad. Sci. U.S.A. 97, 13714-13719.
-
(2000)
Proc. Natl. Acad. Sci. U.S.A.
, vol.97
, pp. 13714-13719
-
-
Wang, B.1
Li, J.2
Xiao, X.3
-
18
-
-
55549113168
-
Construction and analysis of compact muscle-specific promoters for AAV vectors
-
Wang, B., Li, J., Fu, F.H., Chen, C., Zhu, X., Zhou, L., Jiang, X., and Xiao, X. (2008). Construction and analysis of compact muscle-specific promoters for AAV vectors. Gene Ther. 15, 1489-1499.
-
(2008)
Gene Ther.
, vol.15
, pp. 1489-1499
-
-
Wang, B.1
Li, J.2
Fu, F.H.3
Chen, C.4
Zhu, X.5
Zhou, L.6
Jiang, X.7
Xiao, X.8
-
19
-
-
33746874152
-
Adeno-associated virus serotypes: Vector toolkit for human gene therapy
-
Wu, Z., Asokan, A., and Samulski, R.J. (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
-
20
-
-
0029843596
-
Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector
-
Xiao, X., Li, J., and Samulski, R.J. (1996). Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J. Virol. 70, 8098-8108. (Pubitemid 26339120)
-
(1996)
Journal of Virology
, vol.70
, Issue.11
, pp. 8098-8108
-
-
Xiao, X.1
Li, J.2
Samulski, R.J.3
-
21
-
-
62649151797
-
A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection
-
Yang, L., Jiang, J., Drouin, L.M., Agbandje-McKenna, M., Chen, C., Qiao, C., Pu, D., Hu, X., Wang, D.Z., Li, J., and Xiao, X. (2009). A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection. Proc. Natl. Acad. Sci. U.S.A. 106, 3946-3951.
-
(2009)
Proc. Natl. Acad. Sci. U.S.A.
, vol.106
, pp. 3946-3951
-
-
Yang, L.1
Jiang, J.2
Drouin, L.M.3
Agbandje-Mckenna, M.4
Chen, C.5
Qiao, C.6
Pu, D.7
Hu, X.8
Wang, D.Z.9
Li, J.10
Xiao, X.11
-
22
-
-
69249213448
-
A muscle-targeting peptide displayed on AAV2 im-proves muscle tropism on systemic delivery
-
Yu, C.Y., Yuan, Z., Cao, Z., Wang, B., Qiao, C., Li, J., and Xiao, X. (2009). A muscle-targeting peptide displayed on AAV2 im-proves muscle tropism on systemic delivery. Gene Ther. 16, 953-962.
-
(2009)
Gene Ther.
, vol.16
, pp. 953-962
-
-
Yu, C.Y.1
Yuan, Z.2
Cao, Z.3
Wang, B.4
Qiao, C.5
Li, J.6
Xiao, X.7
-
23
-
-
56749104321
-
A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skele-tal muscle transduction in dogs
-
Yue, Y., Ghosh, A., Long, C., Bostick, B., Smith, B.F., Kornegay, J.N., and Duan, D. (2008). A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skele-tal muscle transduction in dogs. Mol. Ther. 16, 1944-1952.
-
(2008)
Mol. Ther.
, vol.16
, pp. 1944-1952
-
-
Yue, Y.1
Ghosh, A.2
Long, C.3
Bostick, B.4
Smith, B.F.5
Kornegay, J.N.6
Duan, D.7
-
24
-
-
34250727017
-
A dual role of EGFR protein tyrosine kinase signaling in ubiquitina-tion of AAV2 capsids and viral second-strand DNA synthesis
-
Zhong, L., Zhao, W., Wu, J., Li, B., Zolotukhin, S., Govindasamy, L., Agbandje-McKenna, M., and Srivastava, A. (2007). A dual role of EGFR protein tyrosine kinase signaling in ubiquitina-tion of AAV2 capsids and viral second-strand DNA synthesis. Mol. Ther. 15, 1323-1330.
-
(2007)
Mol. Ther.
, vol.15
, pp. 1323-1330
-
-
Zhong, L.1
Zhao, W.2
Wu, J.3
Li, B.4
Zolotukhin, S.5
Govindasamy, L.6
Agbandje-Mckenna, M.7
Srivastava, A.8
-
25
-
-
45549090635
-
Next generation of adeno-associated virus 2 vectors: Point muta-tions in tyrosines lead to high-efficiency transduction at lower doses
-
Zhong, L., Li, B., Mah, C.S., Govindasamy, L., Agbandje-McKenna, M., Cooper, M., Herzog, R.W., Zolotukhin, I., Warrington, K.H., Jr., Weigel-Van Aken, K.A., Hobbs, J.A., Zolotukhin, S., Muzyczka, N., and Srivastava, A. (2008). Next generation of adeno-associated virus 2 vectors: Point muta-tions in tyrosines lead to high-efficiency transduction at lower doses. Proc. Natl. Acad. Sci. U.S.A. 105, 7827-7832
-
(2008)
Proc. Natl. Acad. Sci. U.S.A.
, vol.105
, pp. 7827-7832
-
-
Zhong, L.1
Li, B.2
Mah, C.S.3
Govindasamy, L.4
Agbandje-Mckenna, M.5
Cooper, M.6
Herzog, R.W.7
Zolotukhin, I.8
Warrington Jr., K.H.9
Weigel-Van Aken, K.A.10
Hobbs, J.A.11
Zolotukhin, S.12
Muzyczka, N.13
Srivastava, A.14
|