-
1
-
-
84862025459
-
Directed evolution of novel adeno-associated viruses for therapeutic gene delivery
-
Bartel, MA, Weinstein, JR and Schaffer, DV (2012). Directed evolution of novel adeno-associated viruses for therapeutic gene delivery. Gene Ther 19: 694-700.
-
(2012)
Gene Ther
, vol.19
, pp. 694-700
-
-
Ma, B.1
Weinstein, J.R.2
Schaffer, D.V.3
-
2
-
-
32344438754
-
Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
-
Maheshri, N, Koerber, JT, Kaspar, BK and Schaffer, DV (2006). Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat Biotechnol 24: 198-204.
-
(2006)
Nat Biotechnol
, vol.24
, pp. 198-204
-
-
Maheshri, N.1
Koerber, J.T.2
Kaspar, B.K.3
Schaffer, D.V.4
-
3
-
-
32844470411
-
Combinatorial engineering of a gene therapy vector: Directed evolution of adeno-associated virus
-
Perabo, L, Endell, J, King, S, Lux, K, Goldnau, D, Hallek, M et al. (2006). Combinatorial engineering of a gene therapy vector: directed evolution of adeno-associated virus. J Gene Med 8: 155-162.
-
(2006)
J Gene Med
, vol.8
, pp. 155-162
-
-
Perabo, L.1
Endell, J.2
King, S.3
Lux, K.4
Goldnau, D.5
Hallek, M.6
-
4
-
-
84880540708
-
In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous
-
Dalkara, D, Byrne, LC, Klimczak, RR, Visel, M, Yin, L, Merigan, WH et al. (2013). In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous. Sci Transl Med 5: 189ra76.
-
(2013)
Sci Transl Med
, vol.5
, pp. 189ra76
-
-
Dalkara, D.1
Byrne, L.C.2
Klimczak, R.R.3
Visel, M.4
Yin, L.5
Merigan, W.H.6
-
5
-
-
62649151797
-
A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection
-
Yang, L, Jiang, J, Drouin, LM, Agbandje-McKenna, M, Chen, C, Qiao, C et al. (2009). A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection. Proc Natl Acad Sci USA 106: 3946-3951.
-
(2009)
Proc Natl Acad Sci USA
, 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
-
6
-
-
80052401649
-
AAV mediated GDNF secretion from retinal glia slows down retinal degeneration in a rat model of retinitis pigmentosa
-
Dalkara, D, Kolstad, KD, Guerin, KI, Hoffmann, NV, Visel, M, Klimczak, RR et al. (2011). AAV mediated GDNF secretion from retinal glia slows down retinal degeneration in a rat model of retinitis pigmentosa. Mol Ther 19: 1602-1608.
-
(2011)
Mol Ther
, vol.19
, pp. 1602-1608
-
-
Dalkara, D.1
Kolstad, K.D.2
Guerin, K.I.3
Hoffmann, N.V.4
Visel, M.5
Klimczak, R.R.6
-
7
-
-
84894242894
-
Selection and evaluation of clinically relevant AAV variants in a xenograft liver model
-
Lisowski, L, Dane, A P, Chu, K, Zhang, Y, Cunningham, SC, Wilson, EM et al. (2013). Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature 506: 382.
-
(2013)
Nature
, vol.506
, pp. 382
-
-
Lisowski, L.1
Dane, A.P.2
Chu, K.3
Zhang, Y.4
Cunningham, S.C.5
Wilson, E.M.6
-
8
-
-
44249085878
-
Safety and efficacy of gene transfer for Leber's congenital amaurosis
-
Maguire, AM, Simonelli, F, Pierce, EA, Pugh, EN Jr, Mingozzi, F, Bennicelli, J et al. (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, E.N.4
Mingozzi, F.5
Bennicelli, J.6
-
9
-
-
44249120315
-
Effect of gene therapy on visual function in Leber's congenital amaurosis
-
Bainbridge, JW, Smith, AJ, Barker, SS, Robbie, S, Henderson, R, Balaggan, K et al. (2008). Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 358: 2231-2239.
-
(2008)
N Engl J Med
, vol.358
, pp. 2231-2239
-
-
Bainbridge, J.W.1
Smith, A.J.2
Barker, S.S.3
Robbie, S.4
Henderson, R.5
Balaggan, K.6
-
10
-
-
70350620424
-
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: A phase 1 dose-escalation trial
-
Maguire, AM, High, KA, Auricchio, A, Wright, JF, Pierce, EA, Testa, F et al. (2009). Age-dependent effects of RPE65 gene therapy for Leber'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
-
11
-
-
84855161388
-
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
-
Nathwani, AC, Tuddenham, EG, Rangarajan, S, Rosales, C, McIntosh, J, Linch, DC et al. (2011). Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 365: 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
-
12
-
-
57549090220
-
Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients
-
Stroes, ES, Nierman, MC, Meulenberg, JJ, Franssen, R, Twisk, J, Henny, CP et al. (2008). Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients. Arterioscler Thromb Vasc Biol 28: 2303-2304.
-
(2008)
Arterioscler Thromb Vasc Biol
, vol.28
, pp. 2303-2304
-
-
Stroes, E.S.1
Nierman, M.C.2
Meulenberg, J.J.3
Franssen, R.4
Twisk, J.5
Henny, C.P.6
-
13
-
-
77952571307
-
Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency
-
Gaudet, D, de Wal, J, Tremblay, K, Déry, S, van Deventer, S, Freidig, A et al. (2010). Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency. Atheroscler Suppl 11: 55-60.
-
(2010)
Atheroscler Suppl
, vol.11
, pp. 55-60
-
-
Gaudet, D.1
De Wal, J.2
Tremblay, K.3
Déry, S.4
Van Deventer, S.5
Freidig, A.6
-
14
-
-
84860754546
-
Effect of alipogene tiparvovec (AAV1-LPL(S447X)) on postprandial chylomicron metabolism in lipoprotein lipase-deficient patients
-
Carpentier, AC, Frisch, F, Labbé, SM, Gagnon, R, de Wal, J, Greentree, S et al. (2012). Effect of alipogene tiparvovec (AAV1-LPL(S447X)) on postprandial chylomicron metabolism in lipoprotein lipase-deficient patients. J Clin Endocrinol Metab 97: 1635-1644.
-
(2012)
J Clin Endocrinol Metab
, vol.97
, pp. 1635-1644
-
-
Carpentier, A.C.1
Frisch, F.2
Labbé, S.M.3
Gagnon, R.4
De Wal, J.5
Greentree, S.6
-
15
-
-
84897051037
-
Retinal gene therapy in patients with choroideremia: Initial findings from a phase 1/2 clinical trial
-
e-pub ahead of print 16 January 2014
-
Maclaren, RE, Groppe, M, Barnard, AR, Cottriall, CL, Tolmachova, T, Seymour, L et al. (2014). Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. Lancet, e-pub ahead of print 16 January 2014 (doi:10.1016/S0140-6736(13)62117-0).
-
(2014)
Lancet
-
-
Maclaren, R.E.1
Groppe, M.2
Barnard, A.R.3
Cottriall, C.L.4
Tolmachova, T.5
Seymour, L.6
-
16
-
-
34547730702
-
Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/Il2rg-/-mice
-
Azuma, H, Paulk, N, Ranade, A, Dorrell, C, Al-Dhalimy, M, Ellis, E et al. (2007). Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/Il2rg-/-mice. Nat Biotechnol 25: 903-910.
-
(2007)
Nat Biotechnol
, vol.25
, pp. 903-910
-
-
Azuma, H.1
Paulk, N.2
Ranade, A.3
Dorrell, C.4
Al-Dhalimy, M.5
Ellis, E.6
-
17
-
-
77954976233
-
Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: Implications for gene therapy using AAV vectors
-
Boutin, S, Monteilhet, V, Veron, P, Leborgne, C, Benveniste, O, Montus, MF et al. (2010). Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors. Hum Gene Ther 21: 704-712.
-
(2010)
Hum Gene Ther
, vol.21
, pp. 704-712
-
-
Boutin, S.1
Monteilhet, V.2
Veron, P.3
Leborgne, C.4
Benveniste, O.5
Montus, M.F.6
|