-
1
-
-
85020807888
-
-
World Health Organization [accessed March 12, 2017]
-
World Health Organization. Genomic Research Centre: genes and human disease. 2017. Available at: http://www.who.int/genomics/public/geneticdiseases/en/index2.html [accessed March 12, 2017].
-
(2017)
Genomic Research Centre: Genes and Human Disease.
-
-
-
2
-
-
84903587200
-
Engineering adeno-associated viruses for clinical gene therapy
-
Kotterman MA, Schaffer DV. Engineering adeno-associated viruses for clinical gene therapy. Nat Rev Genet 2014;15:445-451.
-
(2014)
Nat Rev Genet
, vol.15
, pp. 445-451
-
-
Kotterman, M.A.1
Schaffer, D.V.2
-
4
-
-
79954622209
-
Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges
-
Mingozzi F, High KA. Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet 2011;12:341-355.
-
(2011)
Nat Rev Genet
, vol.12
, pp. 341-355
-
-
Mingozzi, F.1
High, K.A.2
-
5
-
-
33644820684
-
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
-
Manno CS, Pierce GF, Arruda VR, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006;12:342-348.
-
(2006)
Nat Med
, vol.12
, pp. 342-348
-
-
Manno, C.S.1
Pierce, G.F.2
Arruda, V.R.3
-
6
-
-
84890490382
-
Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: A potential therapy for acute intermittent porphyria
-
Paneda A, Lopez-Franco E, Kaeppel C, et al. Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: a potential therapy for acute intermittent porphyria. Hum Gene Ther 2013; 24:1007-1017.
-
(2013)
Hum Gene Ther
, vol.24
, pp. 1007-1017
-
-
Paneda, A.1
Lopez-Franco, E.2
Kaeppel, C.3
-
7
-
-
84979295330
-
Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria
-
D'Avola D, López-Franco E, Sangro B, et al. Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria. J Hepatol 2016; 65:776-783.
-
(2016)
J Hepatol
, vol.65
, pp. 776-783
-
-
D'Avola, D.1
López-Franco, E.2
Sangro, B.3
-
8
-
-
84954376464
-
Long-term metabolic correction of Wilson's disease in a murine model by gene therapy
-
Murillo O, Moreno-Luqui D, Gazquez C, et al. Long-term metabolic correction of Wilson's disease in a murine model by gene therapy. J Hepatol 2016;64: 419-426.
-
(2016)
J Hepatol
, vol.64
, pp. 419-426
-
-
Murillo, O.1
Moreno-Luqui, D.2
Gazquez, C.3
-
9
-
-
79955581947
-
Phenotypic correction of a mouse model for primary hyperoxaluria with adeno-associated virus gene transfer
-
Salido E, Rodriguez-Pena M, Santana A, et al. Phenotypic correction of a mouse model for primary hyperoxaluria with adeno-associated virus gene transfer. Mol Ther 2011;19:870-875.
-
(2011)
Mol Ther
, vol.19
, pp. 870-875
-
-
Salido, E.1
Rodriguez-Pena, M.2
Santana, A.3
-
10
-
-
84882814879
-
Monogenic diseases that can be cured by liver transplantation
-
Fagiuoli S, Daina E, D'Antiga L, et al. Monogenic diseases that can be cured by liver transplantation. J Hepatol 2013;59:595-612.
-
(2013)
J Hepatol
, vol.59
, pp. 595-612
-
-
Fagiuoli, S.1
Daina, E.2
D'Antiga, L.3
-
11
-
-
84959152780
-
Single point mu-tation in adeno-associated viral vectors: DJ capsid leads to improvement for gene delivery in vivo
-
Mao Y, Wang X, Yan R, et al. Single point mu-tation in adeno-associated viral vectors: DJ capsid leads to improvement for gene delivery in vivo. BMC Biotechnol 2016;16:1-8.
-
(2016)
BMC Biotechnol
, vol.16
, pp. 1-8
-
-
Mao, Y.1
Wang, X.2
Yan, R.3
-
12
-
-
33748943615
-
In-trahepatic injection of adenovirus reduces inflammation and increases gene transfer and therapeutic effect in mice
-
Crettaz J, Berraondo P, Mauleón I, et al. In-trahepatic injection of adenovirus reduces inflammation and increases gene transfer and therapeutic effect in mice. Hepatology 2006;44:623-632.
-
(2006)
Hepatology
, vol.44
, pp. 623-632
-
-
Crettaz, J.1
Berraondo, P.2
Mauleón, I.3
-
13
-
-
84873337445
-
Minimizing the inhibitory effect of neutralizing antibody for efficient gene expression in the liver with adeno-associated virus 8 vectors
-
Mimuro J, Mizukami H, Hishikawa S, et al. Minimizing the inhibitory effect of neutralizing antibody for efficient gene expression in the liver with adeno-associated virus 8 vectors. Mol Ther 2013;21:318-323.
-
(2013)
Mol Ther
, vol.21
, pp. 318-323
-
-
Mimuro, J.1
Mizukami, H.2
Hishikawa, S.3
-
14
-
-
33645912596
-
Im-proved hepatic transduction, reduced systemic vector dissemination, and long-term transgene expression by delivering helper-dependent ade-noviral vectors into the surgically isolated liver of nonhuman primates
-
Brunetti-Pierri N, Ng T, Iannitti DA, et al. Im-proved hepatic transduction, reduced systemic vector dissemination, and long-term transgene expression by delivering helper-dependent ade-noviral vectors into the surgically isolated liver of nonhuman primates. Hum Gene Ther 2006;17: 391-404.
-
(2006)
Hum Gene Ther
, vol.17
, pp. 391-404
-
-
Brunetti-Pierri, N.1
Ng, T.2
Iannitti, D.A.3
-
15
-
-
33947244363
-
Pseudo-hydrodynamic delivery of helper-dependent adenoviral vectors into non-human primates for liver-directed gene therapy
-
Brunetti-Pierri N, Stapleton GE, Palmer DJ, et al. Pseudo-hydrodynamic delivery of helper-dependent adenoviral vectors into non-human primates for liver-directed gene therapy. Mol Ther 2007;15: 732-740.
-
(2007)
Mol Ther
, vol.15
, pp. 732-740
-
-
Brunetti-Pierri, N.1
Stapleton, G.E.2
Palmer, D.J.3
-
16
-
-
63649138339
-
Efficient, Long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates
-
Brunetti-Pierri N, Stapleton GE, Law M, et al. Efficient, Long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates. Mol Ther 2009;17:327-333.
-
(2009)
Mol Ther
, vol.17
, pp. 327-333
-
-
Brunetti-Pierri, N.1
Stapleton, G.E.2
Law, M.3
-
17
-
-
84882343654
-
Transgene expression up to 7 years in nonhuman primates following hepatic transduction with helper-dependent adenoviral vectors
-
Brunetti-Pierri N, Ng T, Iannitti D, et al. Transgene expression up to 7 years in nonhuman primates following hepatic transduction with helper-dependent adenoviral vectors. Hum Gene Ther 2013;24: 761-765.
-
(2013)
Hum Gene Ther
, vol.24
, pp. 761-765
-
-
Brunetti-Pierri, N.1
Ng, T.2
Iannitti, D.3
-
18
-
-
20844460836
-
Increased Hepatic transduction with reduced systemic dissemination and proinflammatory cytokines following hydrodynamic injection of helper-dependent adenoviral vectors
-
Brunetti-Pierri N, Palmer DJ, Mane V, et al. Increased Hepatic transduction with reduced systemic dissemination and proinflammatory cytokines following hydrodynamic injection of helper-dependent adenoviral vectors. Mol Ther 2005;12:99-106.
-
(2005)
Mol Ther
, vol.12
, pp. 99-106
-
-
Brunetti-Pierri, N.1
Palmer, D.J.2
Mane, V.3
-
19
-
-
0032805251
-
Hydrodynamics-based trans-fection in animals by systemic administration of plasmid DNA
-
Liu F, Song Y, Liu D. Hydrodynamics-based trans-fection in animals by systemic administration of plasmid DNA. Gene Ther 1999;6:1258-1266.
-
(1999)
Gene Ther
, vol.6
, pp. 1258-1266
-
-
Liu, F.1
Song, Y.2
Liu, D.3
-
20
-
-
84920538278
-
Gene therapy for rhesus monkeys heterozygous for LDL receptor deficiency by balloon catheter hepatic delivery of helper-dependent adenoviral vector
-
Oka K, Mullins CE, Kushwaha RS, et al. Gene therapy for rhesus monkeys heterozygous for LDL receptor deficiency by balloon catheter hepatic delivery of helper-dependent adenoviral vector. Gene Ther 2014;22:87-95.
-
(2014)
Gene Ther
, vol.22
, pp. 87-95
-
-
Oka, K.1
Mullins, C.E.2
Kushwaha, R.S.3
-
21
-
-
84875917898
-
Comparison of gene transfer to the murine liver following intraperitoneal and intraportal delivery of hepatotropic AAV pseudo-serotypes
-
Dane AP, Wowro SJ, Cunningham SC, et al. Comparison of gene transfer to the murine liver following intraperitoneal and intraportal delivery of hepatotropic AAV pseudo-serotypes. Gene Ther 2013;20:460-464.
-
(2013)
Gene Ther
, vol.20
, pp. 460-464
-
-
Dane, A.P.1
Wowro, S.J.2
Cunningham, S.C.3
|