-
1
-
-
33745250487
-
Analysis of Tumors Arising in Male B6C3F1 Mice with and without AAV Vector Delivery to Liver
-
DOI 10.1016/j.ymthe.2006.03.008, PII S1525001606000918
-
Bell, P., Moscioni, A.D., McCarter, R.J., et al. (2006). Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver. Mol. Ther. 14, 34-44. (Pubitemid 43922140)
-
(2006)
Molecular Therapy
, vol.14
, Issue.1
, pp. 34-44
-
-
Bell, P.1
Moscioni, A.D.2
McCarter, R.J.3
Wu, D.4
Gao, G.5
Hoang, A.6
Sanmiguel, J.C.7
Sun, X.8
Wivel, N.A.9
Raper, S.E.10
Furth, E.E.11
Batshaw, M.L.12
Wilson, J.M.13
-
2
-
-
58849086758
-
Worldwide epidemiology of neutralizing antibodies to adenoassociated viruses
-
Calcedo, R., Vandenberghe, L.H., Gao, G., et al. (2009). Worldwide epidemiology of neutralizing antibodies to adenoassociated viruses. J. Infect. Dis. 199, 381-390.
-
(2009)
J. Infect. Dis
, vol.199
, pp. 381-390
-
-
Calcedo, R.1
Vandenberghe, L.H.2
Gao, G.3
-
3
-
-
33646894965
-
In Vivo Transduction by Intravenous Injection of a Lentiviral Vector Expressing Human ADA into Neonatal ADA Gene Knockout Mice: A Novel Form of Enzyme Replacement Therapy for ADA Deficiency
-
DOI 10.1016/j.ymthe.2006.02.013, PII S1525001606000773
-
Carbonaro, D.A., Jin, X., Petersen, D., et al. (2006). In vivo transduction by intravenous injection of a lentiviral vector expressing human ADA into neonatal ADA gene knockout mice: A novel form of enzyme replacement therapy for ADA deficiency. Mol. Ther. 13, 1110-1120. (Pubitemid 43779186)
-
(2006)
Molecular Therapy
, vol.13
, Issue.6
, pp. 1110-1120
-
-
Carbonaro, D.A.1
Jin, X.2
Petersen, D.3
Wang, X.4
Dorey, F.5
Kil, K.S.6
Aldrich, M.7
Blackburn, M.R.8
Kellems, R.E.9
Kohn, D.B.10
-
4
-
-
77957854750
-
Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction
-
Carrillo-Carrasco, N., Chandler, R.J., Chandrasekaran, S., et al. (2010). Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction. Hum. Gene Ther. 21, 1147-1154.
-
(2010)
Hum. Gene Ther.
, vol.21
, pp. 1147-1154
-
-
Carrillo-Carrasco, N.1
Chandler, R.J.2
Chandrasekaran, S.3
-
5
-
-
74149088239
-
Long-term rescue of a lethal murine model of methylmalonic acidemia using adenoassociated viral gene therapy
-
Chandler, R.J., and Venditti, C.P. (2010). Long-term rescue of a lethal murine model of methylmalonic acidemia using adenoassociated viral gene therapy. Mol. Ther. 18, 11-16.
-
(2010)
Mol. Ther.
, vol.18
, pp. 11-16
-
-
Chandler, R.J.1
Venditti, C.P.2
-
6
-
-
79954533921
-
Adeno-associated virus serotype 8 gene transfer rescues a neonatal lethal murine model of propionic acidemia
-
Chandler, R.J., Chandrasekaran, S., Carrillo-Carrasco, N., et al. (2011). Adeno-associated virus serotype 8 gene transfer rescues a neonatal lethal murine model of propionic acidemia. Hum. Gene Ther. 22, 477-481.
-
(2011)
Hum. Gene Ther.
, vol.22
, pp. 477-481
-
-
Chandler, R.J.1
Chandrasekaran, S.2
Carrillo-Carrasco, N.3
-
7
-
-
79952190655
-
Longterm amelioration of feline mucopolysaccharidosis VI after AAV-mediated liver gene transfer
-
Cotugno, G., Annunziata, P., Tessitore, A., et al. (2011). Longterm amelioration of feline mucopolysaccharidosis VI After AAV-mediated liver gene transfer. Mol. Ther. 19, 461-469.
-
(2011)
Mol. Ther.
, vol.19
, pp. 461-469
-
-
Cotugno, G.1
Annunziata, P.2
Tessitore, A.3
-
8
-
-
44049094812
-
Gene delivery to the juvenile mouse liver using AAV2/8 vectors
-
DOI 10.1038/mt.2008.72, PII MT200872
-
Cunningham, S.C., Dane, A.P., Spinoulas, A., et al. (2008). Gene delivery to the juvenile mouse liver using AAV2/8 vectors. Mol. Ther. 16, 1081-1088. (Pubitemid 351737073)
-
(2008)
Molecular Therapy
, vol.16
, Issue.6
, pp. 1081-1088
-
-
Cunningham, S.C.1
Dane, A.P.2
Spinoulas, A.3
Alexander, I.E.4
-
9
-
-
1542425254
-
AAV-mediated gene transfer to the liver
-
Daly, T.M. (2004). AAV-mediated gene transfer to the liver. Methods Mol. Biol. 246, 195-199.
-
(2004)
Methods Mol. Biol
, vol.246
, pp. 195-199
-
-
Daly, T.M.1
-
10
-
-
0034824239
-
Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer
-
DOI 10.1038/sj.gt.3301420
-
Daly, T.M., Ohlemiller, K.K., Roberts, M.S., et al. (2001). Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer. Gene Ther. 8, 1291-1298. (Pubitemid 32834297)
-
(2001)
Gene Therapy
, vol.8
, Issue.17
, pp. 1291-1298
-
-
Daly, T.M.1
Ohlemiller, K.K.2
Roberts, M.S.3
Vogler, C.A.4
Sands, M.S.5
-
11
-
-
28844483864
-
High levels of persistent expression of α1-Antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses
-
DOI 10.1016/j.ymthe.2005.09.003, PII S1525001605015911
-
De, B.P., Heguy, A., Hackett, N.R., et al. (2006). High levels of persistent expression of a1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses. Mol. Ther. 13, 67-76. (Pubitemid 41763484)
-
(2006)
Molecular Therapy
, vol.13
, Issue.1
, pp. 67-76
-
-
De, B.P.1
Heguy, A.2
Hackett, N.R.3
Ferris, B.4
Leopold, P.L.5
Lee, J.6
Pierre, L.7
Gao, G.8
Wilson, J.M.9
Crystal, R.G.10
-
12
-
-
2642512201
-
Clades of adeno-associated viruses are widely disseminated in human tissues
-
DOI 10.1128/JVI.78.12.6381-6388.2004
-
Gao, G., Vandenberghe, L.H., Alvira, M.R., et al. (2004). Clades of adeno-associated viruses are widely disseminated in human tissues. J. Virol. 78, 6381-6388. (Pubitemid 38715936)
-
(2004)
Journal of Virology
, vol.78
, Issue.12
, 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
-
13
-
-
28844452008
-
Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates
-
DOI 10.1016/j.ymthe.2005.08.017, PII S1525001605015716
-
Gao, G., Lu, Y., Calcedo, R., et al. (2006). Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates. Mol. Ther. 13, 77-87. (Pubitemid 41763485)
-
(2006)
Molecular Therapy
, vol.13
, Issue.1
, pp. 77-87
-
-
Gao, G.1
Lu, Y.2
Calcedo, R.3
Grant, R.L.4
Bell, P.5
Wang, L.6
Figueredo, J.7
Lock, M.8
Wilson, J.M.9
-
14
-
-
0037015049
-
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
-
DOI 10.1073/pnas.182412299
-
Gao, G.P., Alvira, M.R., Wang, L., et al. (2002). Novel adenoassociated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. U.S.A. 99,11854-9. (Pubitemid 34994488)
-
(2002)
Proceedings of the National Academy of Sciences of the United States of America
, vol.99
, Issue.18
, pp. 11854-11859
-
-
Gao, G.-P.1
Alvira, M.R.2
Wang, L.3
Calcedo, R.4
Johnston, J.5
Wilson, J.M.6
-
15
-
-
77952009127
-
Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and cc overexpression
-
Ginn, S.L., Liao, S.H., Dane, A.P., et al. (2010). Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and cc overexpression. Mol. Ther. 18, 965-976.
-
(2010)
Mol. Ther.
, vol.18
, pp. 965-976
-
-
Ginn, S.L.1
Liao, S.H.2
Dane, A.P.3
-
16
-
-
51349090473
-
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
-
Hacein-Bey-Abina, S., Garrigue, A., Wang, G.P., et al. (2008). Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Invest. 118, 3132-3142.
-
(2008)
J. Clin. Invest
, vol.118
, pp. 3132-3142
-
-
Hacein-Bey-Abina, S.1
Garrigue, A.2
Wang, G.P.3
-
17
-
-
77956628142
-
RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy
-
Hu, C., Busuttil, R.W., and Lipshutz, G.S. (2010). RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy. J. Gene Med. 12, 766-778.
-
(2010)
J. Gene Med.
, vol.12
, pp. 766-778
-
-
Hu, C.1
Busuttil, R.W.2
Lipshutz, G.S.3
-
18
-
-
79952122629
-
Neonatal helperdependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII
-
Hu, C., Cela, R.G., Suzuki, M., et al. (2011). Neonatal helperdependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII. Proc. Natl. Acad. Sci. U.S.A. 108, 2082-2087.
-
(2011)
Proc. Natl. Acad. Sci. U.S.A.
, vol.108
, pp. 2082-2087
-
-
Hu, C.1
Cela, R.G.2
Suzuki, M.3
-
19
-
-
17644423927
-
Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector
-
DOI 10.1016/j.ymthe.2004.10.006, PII S152500160401487X
-
Kobayashi, H., Carbonaro, D., Pepper, K., et al. (2005). Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector. Mol. Ther. 11, 776-789. (Pubitemid 40567982)
-
(2005)
Molecular Therapy
, vol.11
, Issue.5
, pp. 776-789
-
-
Kobayashi, H.1
Carbonaro, D.2
Pepper, K.3
Petersen, D.4
Ge, S.5
Jackson, H.6
Shimada, H.7
Moats, R.8
Kohn, D.B.9
-
20
-
-
80053509954
-
Perinatal gene transfer to the liver
-
McKay, T.R., Rahim, A.A., Buckley, S.M., et al. (2011). Perinatal gene transfer to the liver. Curr. Pharm. Des. 17, 2528-2541.
-
(2011)
Curr. Pharm. Des.
, vol.17
, pp. 2528-2541
-
-
McKay, T.R.1
Rahim, A.A.2
Buckley, S.M.3
-
21
-
-
58149459993
-
Enhancing transduction of the liver by adeno-associated viral vectors
-
Nathwani, A.C., Cochrane, M., McIntosh, J., et al. (2009). Enhancing transduction of the liver by adeno-associated viral vectors. Gene Ther. 16, 60-69.
-
(2009)
Gene Ther
, vol.16
, pp. 60-69
-
-
Nathwani, A.C.1
Cochrane, M.2
McIntosh, J.3
-
22
-
-
35348968497
-
Immunology of neonatal gene transfer
-
DOI 10.2174/156652307782151434
-
Ponder, K.P. (2007). Immunology of neonatal gene transfer. Curr. Gene Ther. 7, 403-410. (Pubitemid 47595782)
-
(2007)
Current Gene Therapy
, vol.7
, Issue.5
, pp. 403-410
-
-
Ponder, K.P.1
-
23
-
-
34547101208
-
Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy
-
DOI 10.1038/sj.mt.6300201, PII 6300201
-
Traas, A.M., Wang, P., Ma, X., et al. (2007). Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy. Mol. Ther. 15, 1423-1431. (Pubitemid 47099299)
-
(2007)
Molecular Therapy
, vol.15
, Issue.8
, pp. 1423-1431
-
-
Traas, A.M.1
Wang, P.2
Ma, X.3
Tittiger, M.4
Schaller, L.5
O'donnell, P.6
Sleeper, M.M.7
Vite, C.8
Herati, R.9
Aguirre, G.D.10
Haskins, M.11
Ponder, K.P.12
-
24
-
-
5444252217
-
Fetal and neonatal gene therapy: Benefits and pitfalls
-
DOI 10.1038/sj.gt.3302375
-
Waddington, S.N., Kennea, N.L., Buckley, S.M., et al. (2004). Fetal and neonatal gene therapy: Benefits and pitfalls. Gene Ther. 11(Suppl. 1), S92-S97. (Pubitemid 39359453)
-
(2004)
Gene Therapy
, vol.11
, Issue.SUPPL. 1
-
-
Waddington, S.N.1
Kennea, N.L.2
Buckley, S.M.K.3
Gregory, L.G.4
Themis, M.5
Coutelle, C.6
-
25
-
-
74149091607
-
Systematic evaluation of AAV vectors for liver directed gene transfer in murine models
-
Wang, L., Wang, H., Bell, P., et al. (2010). Systematic evaluation of AAV vectors for liver directed gene transfer in murine models. Mol. Ther. 18, 118-125.
-
(2010)
Mol. Ther.
, vol.18
, pp. 118-125
-
-
Wang, L.1
Wang, H.2
Bell, P.3
-
26
-
-
80455174705
-
AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta
-
Wang, L., Bell, P., Lin, J., et al. (2011a). AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta). Mol. Ther. 19, 2012-2020.
-
(2011)
Mol. Ther
, vol.19
, pp. 2012-2020
-
-
Wang, L.1
Bell, P.2
Lin, J.3
-
27
-
-
80052496589
-
Impact of preexisting immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors
-
Wang, L., Calcedo, R., Bell, P., et al. (2011b). Impact of preexisting immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors. Hum. Gene Ther. 22, 1389-1401.
-
(2011)
Hum. Gene Ther
, vol.22
, pp. 1389-1401
-
-
Wang, L.1
Calcedo, R.2
Bell, P.3
-
28
-
-
84872756393
-
Sustained correction of OTC deficiency in spfash mice using optimized self-complementary AAV2/8 vectors
-
[Epub ahead of print]
-
Wang, L., Wang, H., Morizono, H., et al. (2011c). Sustained correction of OTC deficiency in spfash mice using optimized self-complementary AAV2/8 vectors. Gene Ther. [Epub ahead of print]
-
(2011)
Gene Ther.
-
-
Wang, L.1
Wang, H.2
Morizono, H.3
-
29
-
-
0348038748
-
Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo
-
DOI 10.1038/sj.gt.3302133
-
Wang, Z., Ma, H.I., Li, J., et al. (2003). Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther. 10, 2105-2111. (Pubitemid 37536759)
-
(2003)
Gene Therapy
, vol.10
, Issue.26
, pp. 2105-2111
-
-
Wang, Z.1
Ma, H.-I.2
Li, J.3
Sun, L.4
Zhang, J.5
Xiao, X.6
-
30
-
-
19644378907
-
Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart
-
DOI 10.1038/nbt1073
-
Wang, Z., Zhu, T., Qiao, C., et al. (2005). Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat. Biotechnol. 23, 321-328. (Pubitemid 41094420)
-
(2005)
Nature Biotechnology
, vol.23
, Issue.3
, pp. 321-328
-
-
Wang, Z.1
Zhu, T.2
Qiao, C.3
Zhou, L.4
Wang, B.5
Zhang, J.6
Chen, C.7
Li, J.8
Xiao, X.9
-
31
-
-
77957943865
-
Adenoassociated virus-mediated correction of a canine model of glycogen storage disease type Ia
-
Weinstein, D.A., Correia, C.E., Conlon, T., et al. (2010). Adenoassociated virus-mediated correction of a canine model of glycogen storage disease type Ia. Hum. Gene Ther. 21, 903-910.
-
(2010)
Hum. Gene Ther.
, vol.21
, pp. 903-910
-
-
Weinstein, D.A.1
Correia, C.E.2
Conlon, T.3
-
32
-
-
77953136183
-
Complete normalization of hepatic G6PC deficiency in murine glycogen storage disease type Ia using gene therapy
-
Yiu, W.H., Lee, Y.M., Peng, W.T., et al. (2010). Complete normalization of hepatic G6PC deficiency in murine glycogen storage disease type Ia using gene therapy. Mol. Ther. 18, 1076-1084.
-
(2010)
Mol. Ther.
, vol.18
, pp. 1076-1084
-
-
Yiu, W.H.1
Lee, Y.M.2
Peng, W.T.3
-
33
-
-
10044223213
-
Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors
-
DOI 10.1073/pnas.0407572101
-
Yoshimitsu, M., Sato, T., Tao, K., et al. (2004). Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors. Proc. Natl. Acad. Sci. U.S.A. 101, 16909-16914. (Pubitemid 39601337)
-
(2004)
Proceedings of the National Academy of Sciences of the United States of America
, vol.101
, Issue.48
, pp. 16909-16914
-
-
Yoshimitsu, M.1
Sato, T.2
Tao, K.3
Walia, J.S.4
Rasaiah, V.I.5
Sleep, G.T.6
Murray, G.J.7
Poeppl, A.G.8
Underwood, J.9
West, L.10
Brady, R.O.11
Medin, J.A.12
-
34
-
-
0346336779
-
Neonatal gene transfer with a retroviral vector results in tolerance to human factor IX in mice and dogs
-
DOI 10.1182/blood-2003-06-2181
-
Zhang, J., Xu, L., Haskins, M.E., et al. (2004). Neonatal gene transfer with a retroviral vector results in tolerance to human factor IX in mice and dogs. Blood 103, 143-151. (Pubitemid 38029930)
-
(2004)
Blood
, vol.103
, Issue.1
, pp. 143-151
-
-
Zhang, J.1
Xu, L.2
Haskins, M.E.3
Ponder, K.P.4
|