-
1
-
-
0016682035
-
Food and Drug Administration regulations and licensure
-
Barker, L. F. (1975) Food and Drug Administration regulations and licensure. Fed Proc 34, 1522–1524.
-
(1975)
Fed Proc
, vol.34
, pp. 1522-1524
-
-
Barker, L.F.1
-
2
-
-
21244451129
-
Adeno-associated viral vectors for clinical gene transfer studies
-
Snyder, R. O., and Francis, J. (2005) Adeno-associated viral vectors for clinical gene transfer studies. Curr Gene Ther 5, 311–321.
-
(2005)
Curr Gene Ther
, vol.5
, pp. 311-321
-
-
Snyder, R.O.1
Francis, J.2
-
3
-
-
62449121522
-
Production of Research and Clinical Grade Recombinant Adeno-associated Virus Vectors
-
Berns, K. I., and Flotte, T. R., Eds.), Elsevier, Amsterdam
-
Francis, J. D., and Snyder, R. O. (2005) Production of Research and Clinical Grade Recombinant Adeno-associated Virus Vectors, in Laboratory Techniques in Biochemistry and Molecular Biology (Berns, K. I., and Flotte, T. R., Eds.) pp 19–56, Elsevier, Amsterdam.
-
(2005)
Laboratory Techniques in Biochemistry and Molecular Biology
, pp. 19-56
-
-
Francis, J.D.1
Snyder, R.O.2
-
4
-
-
0029741735
-
Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung
-
Conrad, C. K., Allen, S. S., Afione, S. A., Reynolds, T. C., Beck, S. E., Fee-Maki, M., Barrazza-Ortiz, X., Adams, R., Askin, F. B., Carter, B. J., Guggino, W. B., and Flotte, T. R. (1996) Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung. Gene Ther 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
-
5
-
-
0036764793
-
Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: Safety and immunologic aspects
-
Song, S., Scott-Jorgensen, M., Wang, J., Poirier, A., Crawford, J., Campbell-Thompson, M., and Flotte, T. R. (2002) Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects. Mol Ther 6, 329–335.
-
(2002)
Mol Ther
, vol.6
, pp. 329-335
-
-
Song, S.1
Scott-Jorgensen, M.2
Wang, J.3
Poirier, A.4
Crawford, J.5
Campbell-Thompson, M.6
Flotte, T.R.7
-
6
-
-
20844442874
-
Toxicology and biodistribution studies of a recombinant adeno-associated virus 2-alpha-1 antitrypsin vector
-
Poirier, A., Campbell-Thompson, M., Tang, Q., Scott-Jorgensen, M., Combee, L., Loiler, S., Crawford, J., Song, S., and Flotte, T. R. (2004) Toxicology and biodistribution studies of a recombinant adeno-associated virus 2-alpha-1 antitrypsin vector. Preclinica 2, 43–51.
-
(2004)
Preclinica
, vol.2
, pp. 43-51
-
-
Poirier, A.1
Campbell-Thompson, M.2
Tang, Q.3
Scott-Jorgensen, M.4
Combee, L.5
Loiler, S.6
Crawford, J.7
Song, S.8
Flotte, T.R.9
-
7
-
-
34247270295
-
Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites
-
Flotte, T. R., Conlon, T. J., Poirier, A., Campbell-Thompson, M., and Byrne, B. J. (2007) Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites. Hum Gene Ther 18, 245–256.
-
(2007)
Hum Gene Ther
, vol.18
, pp. 245-256
-
-
Flotte, T.R.1
Conlon, T.J.2
Poirier, A.3
Campbell-Thompson, M.4
Byrne, B.J.5
-
8
-
-
33750591238
-
Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis
-
Jacobson, S. G., Boye, S. L., Aleman, T. S., Conlon, T. J., Zeiss, C. J., Roman, A. J., Cideciyan, A. V., Schwartz, S. B., Komaromy, A. M., Doobrajh, M., Cheung, A. Y., Sumaroka, A., Pearce-Kelling, S. E., Aguirre, G. D., Kaushal, S., Maguire, A. M., Flotte, T. R., and Hauswirth, W. W. (2006) Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. Hum Gene Ther 17, 845–858.
-
(2006)
Hum Gene Ther
, vol.17
, pp. 845-858
-
-
Jacobson, S.G.1
Boye, S.L.2
Aleman, T.S.3
Conlon, T.J.4
Zeiss, C.J.5
Roman, A.J.6
Cideciyan, A.V.7
Schwartz, S.B.8
Komaromy, A.M.9
Doobrajh, M.10
Cheung, A.Y.11
Sumaroka, A.12
Pearce-Kelling, S.E.13
Aguirre, G.D.14
Kaushal, S.15
Maguire, A.M.16
Flotte, T.R.17
Hauswirth, W.W.18
-
9
-
-
33744495152
-
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection
-
Jacobson, S. G., Acland, G. M., Aguirre, G. D., Aleman, T. S., Schwartz, S. B., Cideciyan, A. V., Zeiss, C. J., Komaromy, A. M., Kaushal, S., Roman, A. J., Windsor, E. A., Sumaroka, A., Pearce-Kelling, S. E., Conlon, T. J., Chiodo, V. A., Boye, S. L., Flotte, T. R., Maguire, A. M., Bennett, J., and Hauswirth, W. W. (2006) Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection. Mol Ther 13, 1074–1084.
-
(2006)
Mol Ther
, vol.13
, pp. 1074-1084
-
-
Jacobson, S.G.1
Acland, G.M.2
Aguirre, G.D.3
Aleman, T.S.4
Schwartz, S.B.5
Cideciyan, A.V.6
Zeiss, C.J.7
Komaromy, A.M.8
Kaushal, S.9
Roman, A.J.10
Windsor, E.A.11
Sumaroka, A.12
Pearce-Kelling, S.E.13
Conlon, T.J.14
Chiodo, V.A.15
Boye, S.L.16
Flotte, T.R.17
Maguire, A.M.18
Bennett, J.19
Hauswirth, W.W.20
more..
-
10
-
-
0029091783
-
Transfer of the CFTR gene to the lung of nonhuman primates with E1-deleted, E2a-defective recombinant adenoviruses: A preclinical toxicology study
-
Goldman, M. J., Litzky, L. A., Engelhardt, J. F., and Wilson, J. M. (1995) Transfer of the CFTR gene to the lung of nonhuman primates with E1-deleted, E2a-defective recombinant adenoviruses: a preclinical toxicology study. Hum Gene Ther 6, 839–51.
-
(1995)
Hum Gene Ther
, vol.6
, pp. 839-851
-
-
Goldman, M.J.1
Litzky, L.A.2
Engelhardt, J.F.3
Wilson, J.M.4
-
11
-
-
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, 3227–3334.
-
(1996)
J Virol
, vol.70
, pp. 3227-3334
-
-
Ferrari, F.K.1
Samulski, T.2
Shenk, T.3
Samulski, R.J.4
-
12
-
-
0029655894
-
Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis
-
Fisher, K. J., Gao, G. P., Weitzman, M. D., DeMatteo, R., Burda, J. F., and Wilson, J. M. (1996) Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J Virol 70, 520–532.
-
(1996)
J Virol
, vol.70
, pp. 520-532
-
-
Fisher, K.J.1
Gao, G.P.2
Weitzman, M.D.3
Dematteo, R.4
Burda, J.F.5
Wilson, J.M.6
-
13
-
-
0032711845
-
Delayed expression of adeno-associated virus vector DNA
-
Afione, S. A., Wang, J., Walsh, S., Guggino, W. B., and Flotte, T. R. (1999) Delayed expression of adeno-associated virus vector DNA. Intervirology 42, 213–220.
-
(1999)
Intervirology
, vol.42
, pp. 213-220
-
-
Afione, S.A.1
Wang, J.2
Walsh, S.3
Guggino, W.B.4
Flotte, T.R.5
-
14
-
-
13144305062
-
Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors
-
Song, S., Morgan, M., Ellis, T., Poirier, A., Chesnut, K., Wang, J., Brantly, M., Muzyczka, N., Byrne, B. J., Atkinson, M., and Flotte, T. R. (1998) Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci U S A 95, 14384–14388.
-
(1998)
Proc Natl Acad Sci U S A
, vol.95
, pp. 14384-14388
-
-
Song, S.1
Morgan, M.2
Ellis, T.3
Poirier, A.4
Chesnut, K.5
Wang, J.6
Brantly, M.7
Muzyczka, N.8
Byrne, B.J.9
Atkinson, M.10
Flotte, T.R.11
-
15
-
-
0034882826
-
Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis
-
McCarty, D. M., Monahan, P. E., and Samulski, R. J. (2001) Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther 8, 1248–1254.
-
(2001)
Gene Ther
, vol.8
, pp. 1248-1254
-
-
McCarty, D.M.1
Monahan, P.E.2
Samulski, R.J.3
-
16
-
-
0346777307
-
Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo
-
McCarty, D. M., Fu, H., Monahan, P. E., Toulson, C. E., Naik, P., and Samulski, R. J. (2003) Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther 10, 2112–2118.
-
(2003)
Gene Ther
, vol.10
, pp. 2112-2118
-
-
McCarty, D.M.1
Fu, H.2
Monahan, P.E.3
Toulson, C.E.4
Naik, P.5
Samulski, R.J.6
-
17
-
-
0347286956
-
Self-complementary adeno-associated virus serotype 2 vector: Global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain
-
Fu, H., Muenzer, J., Samulski, R. J., Breese, G., Sifford, J., Zeng, X., and McCarty, D. M. (2003) Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain. Mol Ther 8, 911–917.
-
(2003)
Mol Ther
, vol.8
, pp. 911-917
-
-
Fu, H.1
Muenzer, J.2
Samulski, R.J.3
Breese, G.4
Sifford, J.5
Zeng, X.6
McCarty, D.M.7
-
18
-
-
7044241347
-
Self-complementary adeno-associated virus 2 (AAV)-T cell protein tyrosine phoshatase vectors as helper viruses to improve transduction efficiency of conventional single-stranded AAV vectors in vitro and in vivo
-
Zhong, L., Chen, L., Li, Y., Qing, K., Weigel-Kelley, K. A., Chan, R. J., Yoder, M. C., and Srivastava, A. (2004) Self-complementary adeno-associated virus 2 (AAV)-T cell protein tyrosine phoshatase vectors as helper viruses to improve transduction efficiency of conventional single-stranded AAV vectors in vitro and in vivo. Mol Ther 10, 950–957.
-
(2004)
Mol Ther
, vol.10
, pp. 950-957
-
-
Zhong, L.1
Chen, L.2
Li, Y.3
Qing, K.4
Weigel-Kelley, K.A.5
Chan, R.J.6
Yoder, M.C.7
Srivastava, A.8
-
19
-
-
33746801474
-
Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid
-
Vandenberghe, L. H., Wang, L., Somanathan, S., Zhi, Y., Figueredo, J., Calcedo, R., Sanmiguel, J., Desai, R. A., Chen, C. S., Johnston, J., Grant, R. L., Gao, G., and Wilson, J. M. (2006) Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat Med 12, 967–971.
-
(2006)
Nat Med
, vol.12
, pp. 967-971
-
-
Vandenberghe, L.H.1
Wang, L.2
Somanathan, S.3
Zhi, Y.4
Figueredo, J.5
Calcedo, R.6
Sanmiguel, J.7
Desai, R.A.8
Chen, C.S.9
Johnston, J.10
Grant, R.L.11
Gao, G.12
Wilson, J.M.13
-
20
-
-
18444380309
-
Adeno-associated virus: A ubiquitous commensal of mammals
-
Flotte, T. R., and Berns, K. I. (2005) Adeno-associated virus: a ubiquitous commensal of mammals. Hum Gene Ther 16, 401–407.
-
(2005)
Hum Gene Ther
, vol.16
, pp. 401-407
-
-
Flotte, T.R.1
Berns, K.I.2
-
21
-
-
0031906147
-
Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions
-
Summerford, C., and Samulski, R. J. (1998) Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 72, 1438–1445.
-
(1998)
J Virol
, vol.72
, pp. 1438-1445
-
-
Summerford, C.1
Samulski, R.J.2
-
22
-
-
0032589751
-
AlphaVbeta5 integrin: A co-recep-tor for adeno-associated virus type 2 infection
-
Summerford, C., Bartlett, J. S., and Samulski, R. J. (1999) AlphaVbeta5 integrin: a co-recep-tor for adeno-associated virus type 2 infection. Nat Med 5, 78–82.
-
(1999)
Nat Med
, vol.5
, pp. 78-82
-
-
Summerford, C.1
Bartlett, J.S.2
Samulski, R.J.3
-
23
-
-
0035827546
-
Binding of adeno-associated virus type 5 to 2,3-linked sialic acid is required for gene transfer
-
Walters, R. W., Yi, S. M., Keshavjee, S., Brown, K. E., Welsh, M. J., Chiorini, J. A., and Zabner, J. (2001) Binding of adeno-associated virus type 5 to 2,3-linked sialic acid is required for gene transfer. J Biol Chem 276, 20610–20616.
-
(2001)
J Biol Chem
, vol.276
, pp. 20610-20616
-
-
Walters, R.W.1
Yi, S.M.2
Keshavjee, S.3
Brown, K.E.4
Welsh, M.J.5
Chiorini, J.A.6
Zabner, J.7
-
24
-
-
0033010884
-
Human fibroblast growth factor receptor 1 is a coreceptor for infection by adeno-associated virus 2
-
Qing, K., Mah, C., Hansen, J., Zhou, S., Dwarki, V., and Srivastava, A. (1999) Human fibroblast growth factor receptor 1 is a coreceptor for infection by adeno-associated virus 2. Nat Med 5, 71–77.
-
(1999)
Nat Med
, vol.5
, pp. 71-77
-
-
Qing, K.1
Mah, C.2
Hansen, J.3
Zhou, S.4
Dwarki, V.5
Srivastava, A.6
-
25
-
-
20044374997
-
Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors
-
Chen, S., Kapturczak, M., Loiler, S. A., Zolotukhin, S., Glushakova, O. Y., Madsen, K. M., Samulski, R. J., Hauswirth, W. W., Campbell-Thompson, M., Berns, K. I., Flotte, T. R., Atkinson, M. A., Tisher, C. C., and Agarwal, A. (2005) Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors. Hum Gene Ther 16, 235–247.
-
(2005)
Hum Gene Ther
, vol.16
, pp. 235-247
-
-
Chen, S.1
Kapturczak, M.2
Loiler, S.A.3
Zolotukhin, S.4
Glushakova, O.Y.5
Madsen, K.M.6
Samulski, R.J.7
Hauswirth, W.W.8
Campbell-Thompson, M.9
Berns, K.I.10
Flotte, T.R.11
Atkinson, M.A.12
Tisher, C.C.13
Agarwal, A.14
-
26
-
-
0034091579
-
Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus
-
Duan, D., Yue, Y., Yan, Z., Yang, J., and Engelhardt, J. F. (2000) Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus. J Clin Invest 105, 1573–1587.
-
(2000)
J Clin Invest
, vol.105
, pp. 1573-1587
-
-
Duan, D.1
Yue, Y.2
Yan, Z.3
Yang, J.4
Engelhardt, J.F.5
-
27
-
-
0033799367
-
Endocytosis and nuclear trafficking of adeno-associated virus type 2 are controlled by rac1 and phosphatidylinositol-3 kinase activation
-
Sanlioglu, S., Benson, P. K., Yang, J., Atkinson, E. M., Reynolds, T., and Engelhardt, J. F. (2000) Endocytosis and nuclear trafficking of adeno-associated virus type 2 are controlled by rac1 and phosphatidylinositol-3 kinase activation. J Virol 74, 9184–9196.
-
(2000)
J Virol
, vol.74
, pp. 9184-9196
-
-
Sanlioglu, S.1
Benson, P.K.2
Yang, J.3
Atkinson, E.M.4
Reynolds, T.5
Engelhardt, J.F.6
-
28
-
-
0036171821
-
Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors
-
Yan, Z., Zak, R., Luxton, G. W., Ritchie, T. C., Bantel-Schaal, U., and Engelhardt, J. F. (2002) Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors. J Virol 76, 2043–2053.
-
(2002)
J Virol
, vol.76
, pp. 2043-2053
-
-
Yan, Z.1
Zak, R.2
Luxton, G.W.3
Ritchie, T.C.4
Bantel-Schaal, U.5
Engelhardt, J.F.6
-
29
-
-
1542287505
-
Distinct classes of proteasome-modulating agents cooperatively augment recombinant adeno-associated virus type 2 and type 5-mediated transduction from the apical surfaces of human airway epithelia
-
Yan, Z., Zak, R., Zhang, Y., Ding, W., Godwin, S., Munson, K., Peluso, R., and Engelhardt, J. F. (2004) Distinct classes of proteasome-modulating agents cooperatively augment recombinant adeno-associated virus type 2 and type 5-mediated transduction from the apical surfaces of human airway epithelia. J Virol 78, 2863–74.
-
(2004)
J Virol
, vol.78
, pp. 2863-2874
-
-
Yan, Z.1
Zak, R.2
Zhang, Y.3
Ding, W.4
Godwin, S.5
Munson, K.6
Peluso, R.7
Engelhardt, J.F.8
-
30
-
-
0030058330
-
Recruitment of wild-type and recombinant adeno-associated virus into adenovirus replication centers
-
Weitzman, M. D., Fisher, K. J., and Wilson, J. M. (1996) Recruitment of wild-type and recombinant adeno-associated virus into adenovirus replication centers. J Virol 70, 1845–1854.
-
(1996)
J Virol
, vol.70
, pp. 1845-1854
-
-
Weitzman, M.D.1
Fisher, K.J.2
Wilson, J.M.3
-
31
-
-
0030931601
-
Role of tyrosine phosphorylation of a cellular protein in adeno-associated virus 2-mediated transgene expression
-
Qing, K., Wang, X. S., Kube, D. M., Ponnazhagan, S., Bajpai, A., and Srivastava, A. (1997) Role of tyrosine phosphorylation of a cellular protein in adeno-associated virus 2-mediated transgene expression. Proc Natl Acad Sci U S A 94, 10879–10884.
-
(1997)
Proc Natl Acad Sci U S A
, vol.94
, pp. 10879-10884
-
-
Qing, K.1
Wang, X.S.2
Kube, D.M.3
Ponnazhagan, S.4
Bajpai, A.5
Srivastava, A.6
-
32
-
-
0034849529
-
Adeno-associated virus type 2-mediated gene transfer: Role of cellular FKBP52 protein in transgene expression
-
Qing, K., Hansen, J., Weigel-Kelley, K. A., Tan, M., Zhou, S., and Srivastava, A. (2001) Adeno-associated virus type 2-mediated gene transfer: role of cellular FKBP52 protein in transgene expression. J Virol 75, 8968–8976.
-
(2001)
J Virol
, vol.75
, pp. 8968-8976
-
-
Qing, K.1
Hansen, J.2
Weigel-Kelley, K.A.3
Tan, M.4
Zhou, S.5
Srivastava, A.6
-
33
-
-
0028534879
-
Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration
-
Flotte, T. R., Afione, S. A., and Zeitlin, P. L. (1994) Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration. Am J Respir Cell Mol Biol 11, 517–521.
-
(1994)
Am J Respir Cell Mol Biol
, vol.11
, pp. 517-521
-
-
Flotte, T.R.1
Afione, S.A.2
Zeitlin, P.L.3
-
34
-
-
9244234931
-
In vivo model of adeno-associated virus vector persistence and rescue
-
Afione, S. A., Conrad, C. K., Kearns, W. G., Chunduru, S., Adams, R., Reynolds, T. C., Guggino, W. B., Cutting, G. R., Carter, B. J., and Flotte, T. R. (1996) In vivo model of adeno-associated virus vector persistence and rescue. J Virol 70, 3235–3241.
-
(1996)
J Virol
, vol.70
, pp. 3235-3241
-
-
Afione, S.A.1
Conrad, C.K.2
Kearns, W.G.3
Chunduru, S.4
Adams, R.5
Reynolds, T.C.6
Guggino, W.B.7
Cutting, G.R.8
Carter, B.J.9
Flotte, T.R.10
-
35
-
-
0029836472
-
Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line
-
Kearns, W. G., Afione, S. A., Fulmer, S. B., Pang, M. C., Erikson, D., Egan, M., Landrum, M. J., Flotte, T. R., and Cutting, G. R. (1996) Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line. Gene Ther 3, 748–755.
-
(1996)
Gene Ther
, vol.3
, pp. 748-755
-
-
Kearns, W.G.1
Afione, S.A.2
Fulmer, S.B.3
Pang, M.C.4
Erikson, D.5
Egan, M.6
Landrum, M.J.7
Flotte, T.R.8
Cutting, G.R.9
-
36
-
-
0035957384
-
Effect of DNA-dependent protein kinase on the molecular fate of the rAAV2 genome in skeletal muscle
-
Song, S., Laipis, P. J., Berns, K. I., and Flotte, T. R. (2001) Effect of DNA-dependent protein kinase on the molecular fate of the rAAV2 genome in skeletal muscle. Proceedings of the National Academy of Sciences USA 98, 4084–4088.
-
(2001)
Proceedings of the National Academy of Sciences USA
, vol.98
, pp. 4084-4088
-
-
Song, S.1
Laipis, P.J.2
Berns, K.I.3
Flotte, T.R.4
-
37
-
-
0034960344
-
Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo
-
Nakai, H., Yant, S. R., Storm, T. A., Fuess, S., Meuse, L., and Kay, M. A. (2001) Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J Virol 75, 6969–76.
-
(2001)
J Virol
, vol.75
, pp. 6969-6976
-
-
Nakai, H.1
Yant, S.R.2
Storm, T.A.3
Fuess, S.4
Meuse, L.5
Kay, M.A.6
-
38
-
-
0034993252
-
Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver
-
Chen, Z. Y., Yant, S. R., He, C. Y., Meuse, L., Shen, S., and Kay, M. A. (2001) Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver. Mol Ther 3, 403–410.
-
(2001)
Mol Ther
, vol.3
, pp. 403-410
-
-
Chen, Z.Y.1
Yant, S.R.2
He, C.Y.3
Meuse, L.4
Shen, S.5
Kay, M.A.6
-
39
-
-
1242319264
-
DNA-dependent PK inhibits adeno-associated virus DNA integration
-
Song, S., Lu, Y., Choi, Y. K., Han, Y., Tang, Q., Zhao, G., Berns, K. I., and Flotte, T. R. (2004) DNA-dependent PK inhibits adeno-associated virus DNA integration. Proc Natl Acad Sci USA 101, 2112–2116.
-
(2004)
Proc Natl Acad Sci USA
, vol.101
, pp. 2112-2116
-
-
Song, S.1
Lu, Y.2
Choi, Y.K.3
Han, Y.4
Tang, Q.5
Zhao, G.6
Berns, K.I.7
Flotte, T.R.8
-
40
-
-
0037383480
-
Consequences of DNA-dependent protein kinase catalytic subunit deficiency on recombinant adeno-associated virus genome circular-ization and heterodimerization in muscle tissue
-
Duan, D., Yue, Y., and Engelhardt, J. F. (2003) Consequences of DNA-dependent protein kinase catalytic subunit deficiency on recombinant adeno-associated virus genome circular-ization and heterodimerization in muscle tissue. J Virol 77, 4751–4759.
-
(2003)
J Virol
, vol.77
, pp. 4751-4759
-
-
Duan, D.1
Yue, Y.2
Engelhardt, J.F.3
-
41
-
-
0034612248
-
Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy
-
Yan, Z., Zhang, Y., Duan, D., and Engelhardt, J. F. (2000) Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy. Proc Natl Acad Sci USA 97, 6716–6721.
-
(2000)
Proc Natl Acad Sci USA
, vol.97
, pp. 6716-6721
-
-
Yan, Z.1
Zhang, Y.2
Duan, D.3
Engelhardt, J.F.4
-
42
-
-
10744227749
-
Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: A multicenter, double-blind, placebo-controlled trial
-
Moss, R. B., Rodman, D., Spencer, L. T., Aitken, M. L., Zeitlin, P. L., Waltz, D., Milla, C., Brody, A. S., Clancy, J. P., Ramsey, B., Hamblett, N., and Heald, A. E. (2004) Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial. Chest 125, 509–521.
-
(2004)
Chest
, vol.125
, pp. 509-521
-
-
Moss, R.B.1
Rodman, D.2
Spencer, L.T.3
Aitken, M.L.4
Zeitlin, P.L.5
Waltz, D.6
Milla, C.7
Brody, A.S.8
Clancy, J.P.9
Ramsey, B.10
Hamblett, N.11
Heald, A.E.12
-
43
-
-
0032853032
-
Repeated delivery of adeno-associated virus vectors to the rabbit airway
-
Beck, S. E., Jones, L. A., Chesnut, K., Walsh, S. M., Reynolds, T. C., Carter, B. J., Askin, F. B., Flotte, T. R., and Guggino, W. B. (1999) Repeated delivery of adeno-associated virus vectors to the rabbit airway. J Virol 73, 9446–9455.
-
(1999)
J Virol
, vol.73
, pp. 9446-9455
-
-
Beck, S.E.1
Jones, L.A.2
Chesnut, K.3
Walsh, S.M.4
Reynolds, T.C.5
Carter, B.J.6
Askin, F.B.7
Flotte, T.R.8
Guggino, W.B.9
-
44
-
-
0033960160
-
Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes
-
Halbert, C. L., Rutledge, E. A., Allen, J. M., Russell, D. W., and Miller, A. D. (2000) Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes. J Virol 74, 1524–1532.
-
(2000)
J Virol
, vol.74
, pp. 1524-1532
-
-
Halbert, C.L.1
Rutledge, E.A.2
Allen, J.M.3
Russell, D.W.4
Miller, A.D.5
-
45
-
-
70349481529
-
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
-
Brantly, M. L., Chulay, J. D., Wang, L., Mueller, C., Humphries, M., Spencer, L. T., Rouhani, F., Conlon, T. J., Calcedo, R., Betts, M. R., Spencer, C., Byrne, B. J., Wilson, J. M., and Flotte, T. R. (2009) Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc Natl Acad Sci U S A 106, 16363–16368.
-
(2009)
Proc Natl Acad Sci U S A
, vol.106
, pp. 16363-16368
-
-
Brantly, M.L.1
Chulay, J.D.2
Wang, L.3
Mueller, C.4
Humphries, M.5
Spencer, L.T.6
Rouhani, F.7
Conlon, T.J.8
Calcedo, R.9
Betts, M.R.10
Spencer, C.11
Byrne, B.J.12
Wilson, J.M.13
Flotte, T.R.14
-
46
-
-
34147098413
-
CD8(+) T-cell responses to adeno-associated virus capsid in humans
-
Mingozzi, F., Maus, M. V., Hui, D. J., Sabatino, D. E., Murphy, S. L., Rasko, J. E., Ragni, M. V., Manno, C. S., Sommer, J., Jiang, H., Pierce, G. F., Ertl, H. C., and High, K. A. (2007) CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nat Med 13, 419–422.
-
(2007)
Nat Med
, vol.13
, 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.6
Ragni, M.V.7
Manno, C.S.8
Sommer, J.9
Jiang, H.10
Pierce, G.F.11
Ertl, H.C.12
High, K.A.13
-
47
-
-
0032826773
-
Latent adeno-associated virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model
-
Hernandez, Y. J., Wang, J., Kearns, W. G., Loiler, S., Poirier, A., and Flotte, T. R. (1999) Latent adeno-associated virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model. J Virol 73, 8549–8558.
-
(1999)
J Virol
, vol.73
, pp. 8549-8558
-
-
Hernandez, Y.J.1
Wang, J.2
Kearns, W.G.3
Loiler, S.4
Poirier, A.5
Flotte, T.R.6
-
48
-
-
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., Dake, M., Kaye, R., Razavi, M., Zajko, A., Zehnder, J., Rustagi, P. K., Nakai, H., Chew, A., Leonard, D., Wright, J. F., Lessard, R. R., Sommer, J. M., Tigges, M., Sabatino, D., Luk, A., Jiang, H., Mingozzi, F., Couto, L., Ertl, H. C., High, K. A., and Kay, M. A. (2006) Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 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..
-
49
-
-
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., and Jacobson, S. G. (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
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
-
50
-
-
54449085219
-
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
-
Cideciyan, A. V., Aleman, T. S., Boye, S. L., Schwartz, S. B., Kaushal, S., Roman, A. J., Pang, J. J., Sumaroka, A., Windsor, E. A., Wilson, J. M., Flotte, T. R., Fishman, G. A., Heon, E., Stone, E. M., Byrne, B. J., Jacobson, S. G., and Hauswirth, W. W. (2008) Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A 105, 15112–15117.
-
(2008)
Proc Natl Acad Sci U S A
, vol.105
, pp. 15112-15117
-
-
Cideciyan, A.V.1
Aleman, T.S.2
Boye, S.L.3
Schwartz, S.B.4
Kaushal, S.5
Roman, A.J.6
Pang, J.J.7
Sumaroka, A.8
Windsor, E.A.9
Wilson, J.M.10
Flotte, T.R.11
Fishman, G.A.12
Heon, E.13
Stone, E.M.14
Byrne, B.J.15
Jacobson, S.G.16
Hauswirth, W.W.17
-
51
-
-
77649272865
-
Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway
-
Flotte, T. R., Fischer, A. C., Goetzmann, J., Mueller, C., Cebotaru, L., Yan, Z., Wang, L., Wilson, J. M., Guggino, W. B., and Engelhardt, J. F. (2009) Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway. Mol Ther 18, 594–600.
-
(2009)
Mol Ther
, vol.18
, pp. 594-600
-
-
Flotte, T.R.1
Fischer, A.C.2
Goetzmann, J.3
Mueller, C.4
Cebotaru, L.5
Yan, Z.6
Wang, L.7
Wilson, J.M.8
Guggino, W.B.9
Engelhardt, J.F.10
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