-
1
-
-
79960649903
-
Gene therapy for haemophilia: A long and winding road
-
High, KA (2011). Gene therapy for haemophilia: a long and winding road. J Thromb Haemost 9 (suppl. 1): 2-11.
-
(2011)
J Thromb Haemost
, vol.9
, Issue.SUPPL. 1
, pp. 2-11
-
-
High, K.A.1
-
2
-
-
79952149008
-
Hemophilia gene therapy: A Holy Grail found
-
Ponder, KP (2011). Hemophilia gene therapy: a Holy Grail found. Mol Ther 19: 427-428.
-
(2011)
Mol Ther
, vol.19
, pp. 427-428
-
-
Ponder, K.P.1
-
3
-
-
33947218846
-
Immune responses to AAV capsid: Are mice not humans after all?
-
DOI 10.1038/sj.mt.6300123, PII 6300123
-
Herzog, RW (2007). Immune responses to AAV capsid: are mice not humans after all? Mol Ther 15: 649-650. (Pubitemid 46431983)
-
(2007)
Molecular Therapy
, vol.15
, Issue.4
, pp. 649-650
-
-
Herzog, R.W.1
-
4
-
-
80455174048
-
Capsid-specifc T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primates
-
Li, H, Lasaro, MO, Jia, B, Lin, SW, Haut, LH, High, KA et al. (2011). Capsid-specifc T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primates. Mol Ther 19: 2021-2030.
-
(2011)
Mol Ther
, vol.19
, pp. 2021-2030
-
-
Li, H.1
Lasaro, M.O.2
Jia, B.3
Lin, S.W.4
Haut, L.H.5
High, K.A.6
-
5
-
-
80455164698
-
Systemic administration of AAV8-a-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression
-
Nietupski, JB, Hurlbut, GD, Ziegler, RJ, Chu, Q, Hodges, BL, Ashe, KM et al. (2011). Systemic administration of AAV8-a-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression. Mol Ther 19: 1999-2011.
-
(2011)
Mol Ther
, vol.19
, pp. 1999-2011
-
-
Nietupski, J.B.1
Hurlbut, G.D.2
Ziegler, R.J.3
Chu, Q.4
Hodges, B.L.5
Ashe, K.M.6
-
6
-
-
80455174705
-
AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta
-
Wang, L, Bell, P, Lin, J, Calcedo, R, Tarantal, AF and Wilson, JM (2011). 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
Calcedo, R.4
Tarantal, A.F.5
Wilson, J.M.6
-
7
-
-
80455174043
-
Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques
-
Mattar, CNZ, Nathwani, AC, Waddington, SN, Dighe, N, Kaeppel, C, Nowrouzi, A et al. (2011). Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques. Mol Ther 19: 1950-1960.
-
(2011)
Mol Ther
, vol.19
, pp. 1950-1960
-
-
Cnz, M.1
Nathwani, A.C.2
Waddington, S.N.3
Dighe, N.4
Kaeppel, C.5
Nowrouzi, A.6
-
8
-
-
78049478714
-
Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy
-
Hurlbut, GD, Ziegler, RJ, Nietupski, JB, Foley, JW, Woodworth, LA, Meyers, E et al. (2010). Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy. Mol Ther 18: 1983-1994.
-
(2010)
Mol Ther
, vol.18
, pp. 1983-1994
-
-
Hurlbut, G.D.1
Ziegler, R.J.2
Nietupski, J.B.3
Foley, J.W.4
Woodworth, L.A.5
Meyers, E.6
-
9
-
-
74149088098
-
The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques
-
Wang, L, Calcedo, R, Wang, H, Bell, P, Grant, R, Vandenberghe, LH et al. (2010). The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques. Mol Ther 18: 126-134.
-
(2010)
Mol Ther
, vol.18
, pp. 126-134
-
-
Wang, L.1
Calcedo, R.2
Wang, H.3
Bell, P.4
Grant, R.5
Vandenberghe, L.H.6
-
10
-
-
33846934410
-
Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates
-
DOI 10.1182/blood-2006-03-010181
-
Nathwani, AC, Gray, JT, McIntosh, J, Ng, CYC, Zhou, J, Spence, S et al. (2007). Safe and effcient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates. Blood 109: 1414-1421. (Pubitemid 46239572)
-
(2007)
Blood
, vol.109
, Issue.4
, pp. 1414-1421
-
-
Nathwani, A.C.1
Gray, J.T.2
McIntosh, J.3
Ng, C.Y.C.4
Zhou, J.5
Spence, Y.6
Cochrane, M.7
Gray, E.8
Tuddenham, E.G.D.9
Davidoff, A.M.10
-
11
-
-
79955597265
-
Long-term safety and effcacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins
-
Nathwani, AC, Rosales, C, McIntosh, J, Rastegarlari, G, Nathwani, D, Raj, D et al. (2011). Long-term safety and effcacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol Ther 19: 876-885.
-
(2011)
Mol Ther
, vol.19
, pp. 876-885
-
-
Nathwani, A.C.1
Rosales, C.2
McIntosh, J.3
Rastegarlari, G.4
Nathwani, D.5
Raj, D.6
-
12
-
-
80052497157
-
Adeno-associated virus antibody profles in newborns, children and adolescents
-
Calcedo, R, Morizono, H, Wang, L, McCarter, R, He, J, Jones, D et al. (2011). Adeno-associated virus antibody profles in newborns, children and adolescents. Clin Vaccine Immunol 18: 1586-1588.
-
(2011)
Clin Vaccine Immunol
, vol.18
, pp. 1586-1588
-
-
Calcedo, R.1
Morizono, H.2
Wang, L.3
McCarter, R.4
He, J.5
Jones, D.6
-
13
-
-
77953146507
-
Hepatic AAV gene transfer and the immune system: Friends or foes?
-
Herzog, RW (2010). Hepatic AAV gene transfer and the immune system: friends or foes? Mol Ther 18: 1063-1066.
-
(2010)
Mol Ther
, vol.18
, pp. 1063-1066
-
-
Herzog, R.W.1
|