-
2
-
-
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, et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet 2009; 374: 1597-1605.
-
(2009)
Lancet
, vol.374
, pp. 1597-1605
-
-
Maguire, A.M.1
High, K.A.2
Auricchio, A.3
-
3
-
-
84855161388
-
Adenovirus-associated virus vector-mediated gene transfer in haemophilia B
-
Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in haemophilia B. N Engl J Med 2011; 365: 2357-2365.
-
(2011)
N Engl J Med
, vol.365
, pp. 2357-2365
-
-
Nathwani, A.C.1
Tuddenham, E.G.2
Rangarajan, S.3
-
4
-
-
77954245040
-
Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for haemophilia B
-
Arruda VR, Stedman HH, Haurigot V, et al. Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for haemophilia B. Blood 2010; 115: 4678-4688.
-
(2010)
Blood
, vol.115
, pp. 4678-4688
-
-
Arruda, V.R.1
Stedman, H.H.2
Haurigot, V.3
-
5
-
-
84856296022
-
AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous haemorrhage
-
Binny C, McIntosh J, Della PM, et al. AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous haemorrhage. Blood 2012; 119: 957-966.
-
(2012)
Blood
, vol.119
, pp. 957-966
-
-
Binny, C.1
McIntosh, J.2
Della, P.M.3
-
6
-
-
59649122961
-
Long-term correction of inhibitor-prone haemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy
-
Niemeyer GP, Herzog RW, Mount J, et al. Long-term correction of inhibitor-prone haemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy. Blood 2009; 113: 797-806.
-
(2009)
Blood
, vol.113
, pp. 797-806
-
-
Niemeyer, G.P.1
Herzog, R.W.2
Mount, J.3
-
7
-
-
54249126862
-
Gene therapy using adeno-associated virus vectors
-
Daya S, Berns KI. Gene therapy using adeno-associated virus vectors. Clin Microbiol Rev 2008; 21: 583-593.
-
(2008)
Clin Microbiol Rev
, vol.21
, pp. 583-593
-
-
Daya, S.1
Berns, K.I.2
-
8
-
-
0035032662
-
Gene therapy restores vision in a canine model of childhood blindness
-
Acland GM, Aguirre GD, Ray J, et al. Gene therapy restores vision in a canine model of childhood blindness. Nat Genet 2001; 28: 92-95.
-
(2001)
Nat Genet
, vol.28
, pp. 92-95
-
-
Acland, G.M.1
Aguirre, G.D.2
Ray, J.3
-
9
-
-
10044240371
-
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
-
Goyenvalle A, Vulin A, Fougerousse F, et al. Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science 2004; 306: 1796-1799.
-
(2004)
Science
, vol.306
, pp. 1796-1799
-
-
Goyenvalle, A.1
Vulin, A.2
Fougerousse, F.3
-
10
-
-
4544326444
-
Long-term correction of murine lipoprotein lipase deficiency with AAV1-mediated gene transfer of the naturally occurring LPL(S447X) beneficial mutation
-
Ross CJ, Twisk J, Meulenberg JM, et al. Long-term correction of murine lipoprotein lipase deficiency with AAV1-mediated gene transfer of the naturally occurring LPL(S447X) beneficial mutation. Hum Gene Ther 2004; 15: 906-919.
-
(2004)
Hum Gene Ther
, vol.15
, pp. 906-919
-
-
Ross, C.J.1
Twisk, J.2
Meulenberg, J.M.3
-
11
-
-
33646933211
-
Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation
-
Ross CJ, Twisk J, Bakker AC, et al. Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation. Hum Gene Ther 2006; 17: 487-499.
-
(2006)
Hum Gene Ther
, vol.17
, pp. 487-499
-
-
Ross, C.J.1
Twisk, J.2
Bakker, A.C.3
-
12
-
-
77952571307
-
Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency
-
Gaudet D, de Wal J, Tremblay K, et al. Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency. Atheroscler Suppl 2010; 11: 55-60.
-
(2010)
Atheroscler Suppl
, vol.11
, pp. 55-60
-
-
Gaudet, D.1
de Wal, J.2
Tremblay, K.3
-
13
-
-
79955597265
-
Long-term safety and efficacy 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, et al. Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol Ther 2011; 19: 876-885.
-
(2011)
Mol Ther
, vol.19
, pp. 876-885
-
-
Nathwani, A.C.1
Rosales, C.2
McIntosh, J.3
-
14
-
-
84866762773
-
Gene therapy for muscular dystrophy: lessons learned and path forward
-
Mendell JR, Rodino-Klapac L, Sahenk Z, et al. Gene therapy for muscular dystrophy: lessons learned and path forward. Neurosci Lett 2012; 527: 90-99.
-
(2012)
Neurosci Lett
, vol.527
, pp. 90-99
-
-
Mendell, J.R.1
Rodino-Klapac, L.2
Sahenk, Z.3
-
15
-
-
34547514608
-
Muscle as a target for supplementary factor IX gene transfer
-
Hoffman BE, Dobrzynski E, Wang L, et al. Muscle as a target for supplementary factor IX gene transfer. Hum Gene Ther 2007; 18: 603-613.
-
(2007)
Hum Gene Ther
, vol.18
, pp. 603-613
-
-
Hoffman, B.E.1
Dobrzynski, E.2
Wang, L.3
-
16
-
-
0030451214
-
Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein
-
Kessler PD, Podsakoff GM, Chen X, et al. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA 1996; 93: 14082-14087.
-
(1996)
Proc Natl Acad Sci USA
, vol.93
, pp. 14082-14087
-
-
Kessler, P.D.1
Podsakoff, G.M.2
Chen, X.3
-
17
-
-
0030055516
-
Investigation of chronic hepatitis C infection in individuals with haemophilia: assessment of invasive and non-invasive methods
-
Hanley JP, Jarvis LM, Andrews J, et al. Investigation of chronic hepatitis C infection in individuals with haemophilia: assessment of invasive and non-invasive methods. Br J Haematol 1996; 94: 159-165.
-
(1996)
Br J Haematol
, vol.94
, pp. 159-165
-
-
Hanley, J.P.1
Jarvis, L.M.2
Andrews, J.3
-
18
-
-
77954244683
-
Safety of AAV factor IX peripheral transvenular gene delivery to muscle in haemophilia B dogs
-
Haurigot V, Mingozzi F, Buchlis G, et al. Safety of AAV factor IX peripheral transvenular gene delivery to muscle in haemophilia B dogs. Mol Ther 2010; 18: 1318-1329.
-
(2010)
Mol Ther
, vol.18
, pp. 1318-1329
-
-
Haurigot, V.1
Mingozzi, F.2
Buchlis, G.3
-
19
-
-
0013442744
-
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe haemophilia B
-
Manno CS, Chew AJ, Hutchison S, et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe haemophilia B. Blood 2003; 101: 2963-2972.
-
(2003)
Blood
, vol.101
, pp. 2963-2972
-
-
Manno, C.S.1
Chew, A.J.2
Hutchison, S.3
-
20
-
-
63049086019
-
When to treat and the benefits of treating hepatitis C in patients with haemophilia
-
Patel H, Heathcote EJ. When to treat and the benefits of treating hepatitis C in patients with haemophilia. Haemophilia 2009; 15: 20-32.
-
(2009)
Haemophilia
, vol.15
, pp. 20-32
-
-
Patel, H.1
Heathcote, E.J.2
-
21
-
-
0035877984
-
Factors influencing the development of an anti-factor IX (FIX) immune response following administration of adeno-associated virus-FIX
-
Ge Y, Powell S, Van RM, McArthur JG. Factors influencing the development of an anti-factor IX (FIX) immune response following administration of adeno-associated virus-FIX. Blood 2001; 97: 3733-3737.
-
(2001)
Blood
, vol.97
, pp. 3733-3737
-
-
Ge, Y.1
Powell, S.2
Van, R.M.3
McArthur, J.G.4
-
22
-
-
21244487991
-
Major role of local immune responses in antibody formation to factor IX in AAV gene transfer
-
Wang L, Cao O, Swalm B, et al. Major role of local immune responses in antibody formation to factor IX in AAV gene transfer. Gene Ther 2005; 12: 1453-1464.
-
(2005)
Gene Ther
, vol.12
, pp. 1453-1464
-
-
Wang, L.1
Cao, O.2
Swalm, B.3
-
23
-
-
33845425578
-
Induction of robust immune responses against human immunodeficiency virus is supported by the inherent tropism of adeno-associated virus type 5 for dendritic cells
-
Xin KQ, Mizukami H, Urabe M, et al. Induction of robust immune responses against human immunodeficiency virus is supported by the inherent tropism of adeno-associated virus type 5 for dendritic cells. J Virol 2006; 80: 11899-11910.
-
(2006)
J Virol
, vol.80
, pp. 11899-11910
-
-
Xin, K.Q.1
Mizukami, H.2
Urabe, M.3
-
24
-
-
70350125101
-
Distinct immune responses to transgene products from rAAV1 and rAAV8 vectors
-
Lu Y, Song S. Distinct immune responses to transgene products from rAAV1 and rAAV8 vectors. Proc Natl Acad Sci USA 2009; 106: 17158-17162.
-
(2009)
Proc Natl Acad Sci USA
, vol.106
, pp. 17158-17162
-
-
Lu, Y.1
Song, S.2
-
25
-
-
62549094869
-
Transduction of nonhuman primate brain with adeno-associated virus serotype 1: vector trafficking and immune response
-
Hadaczek P, Forsayeth J, Mirek H, et al. Transduction of nonhuman primate brain with adeno-associated virus serotype 1: vector trafficking and immune response. Hum Gene Ther 2009; 20: 225-237.
-
(2009)
Hum Gene Ther
, vol.20
, pp. 225-237
-
-
Hadaczek, P.1
Forsayeth, J.2
Mirek, H.3
-
26
-
-
19344366672
-
Systemic protein delivery by muscle-gene transfer is limited by a local immune response
-
Wang L, Dobrzynski E, Schlachterman A, Cao O, Herzog RW. Systemic protein delivery by muscle-gene transfer is limited by a local immune response. Blood 2005; 105: 4226-4234.
-
(2005)
Blood
, vol.105
, pp. 4226-4234
-
-
Wang, L.1
Dobrzynski, E.2
Schlachterman, A.3
Cao, O.4
Herzog, R.W.5
-
27
-
-
77957725001
-
Dystrophin immunity in Duchenne's muscular dystrophy
-
Mendell JR, Campbell K, Rodino-Klapac L, et al. Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med 2010; 363: 1429-1437.
-
(2010)
N Engl J Med
, vol.363
, pp. 1429-1437
-
-
Mendell, J.R.1
Campbell, K.2
Rodino-Klapac, L.3
-
28
-
-
0036892308
-
Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product
-
Yuasa K, Sakamoto M, Miyagoe-Suzuki Y, et al. Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product. Gene Ther 2002; 9: 1576-1588.
-
(2002)
Gene Ther
, vol.9
, pp. 1576-1588
-
-
Yuasa, K.1
Sakamoto, M.2
Miyagoe-Suzuki, Y.3
-
29
-
-
84862158942
-
Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates
-
Unzu C, Hervas-Stubbs S, Sampedro A, et al. Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates. J Transl Med 2012; 10: 122.
-
(2012)
J Transl Med
, vol.10
, pp. 122
-
-
Unzu, C.1
Hervas-Stubbs, S.2
Sampedro, A.3
-
30
-
-
69949184405
-
Strategies to modulate immune responses: a new frontier for gene therapy
-
Arruda VR, Favaro P, Finn JD. Strategies to modulate immune responses: a new frontier for gene therapy. Mol Ther 2009; 17: 1492-1503.
-
(2009)
Mol Ther
, vol.17
, pp. 1492-1503
-
-
Arruda, V.R.1
Favaro, P.2
Finn, J.D.3
-
31
-
-
37349073878
-
Infection in solid-organ transplant recipients
-
Fishman JA. Infection in solid-organ transplant recipients. N Engl J Med 2007; 357: 2601-2614.
-
(2007)
N Engl J Med
, vol.357
, pp. 2601-2614
-
-
Fishman, J.A.1
-
32
-
-
0035966319
-
microRNAs: tiny regulators with great potential
-
Ambros V. microRNAs: tiny regulators with great potential. Cell 2001; 107: 823-826.
-
(2001)
Cell
, vol.107
, pp. 823-826
-
-
Ambros, V.1
-
33
-
-
1942435249
-
Micromanagers of gene expression: the potentially widespread influence of metazoan microRNAs
-
Bartel DP, Chen CZ. Micromanagers of gene expression: the potentially widespread influence of metazoan microRNAs. Nat Rev Genet 2004; 5: 396-400.
-
(2004)
Nat Rev Genet
, vol.5
, pp. 396-400
-
-
Bartel, D.P.1
Chen, C.Z.2
-
34
-
-
0346727524
-
MicroRNAs modulate haematopoietic lineage differentiation
-
Chen CZ, Li L, Lodish HF, Bartel DP. MicroRNAs modulate haematopoietic lineage differentiation. Science 2004; 303: 83-86.
-
(2004)
Science
, vol.303
, pp. 83-86
-
-
Chen, C.Z.1
Li, L.2
Lodish, H.F.3
Bartel, D.P.4
-
35
-
-
0037197803
-
Identification of tissue-specific microRNAs from mouse
-
Lagos-Quintana M, Rauhut R, Yalcin A, et al. Identification of tissue-specific microRNAs from mouse. Curr Biol 2002; 12: 735-739.
-
(2002)
Curr Biol
, vol.12
, pp. 735-739
-
-
Lagos-Quintana, M.1
Rauhut, R.2
Yalcin, A.3
-
36
-
-
33646564701
-
Endogenous microRNA regulation suppresses transgene expression in haematopoietic lineages and enables stable gene transfer
-
Brown BD, Venneri MA, Zingale A, Sergi SL, Naldini L. Endogenous microRNA regulation suppresses transgene expression in haematopoietic lineages and enables stable gene transfer. Nat Med 2006; 12: 585-591.
-
(2006)
Nat Med
, vol.12
, pp. 585-591
-
-
Brown, B.D.1
Venneri, M.A.2
Zingale, A.3
Sergi, S.L.4
Naldini, L.5
-
37
-
-
73949157699
-
In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunological tolerance
-
Annoni A, Brown BD, Cantore A, et al. In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunological tolerance. Blood 2009; 114: 5152-5161.
-
(2009)
Blood
, vol.114
, pp. 5152-5161
-
-
Annoni, A.1
Brown, B.D.2
Cantore, A.3
-
38
-
-
79953322192
-
A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of haemophilia A
-
Matsui H, Hegadorn C, Ozelo M, et al. A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of haemophilia A. Mol Ther 2011; 19: 723-730.
-
(2011)
Mol Ther
, vol.19
, pp. 723-730
-
-
Matsui, H.1
Hegadorn, C.2
Ozelo, M.3
-
39
-
-
0036431701
-
Insect cells as a factory to produce adeno-associated virus type 2 vectors
-
Urabe M, Ding C, Kotin RM. Insect cells as a factory to produce adeno-associated virus type 2 vectors. Hum Gene Ther 2002; 13: 1935-43.
-
(2002)
Hum Gene Ther
, vol.13
, pp. 1935-1943
-
-
Urabe, M.1
Ding, C.2
Kotin, R.M.3
-
40
-
-
0031058024
-
Recombinant adeno-associated virus for muscle directed gene therapy
-
Fisher KJ, Jooss K, Alston J, et al. Recombinant adeno-associated virus for muscle directed gene therapy. Nat Med 1997; 3: 306-312.
-
(1997)
Nat Med
, vol.3
, pp. 306-312
-
-
Fisher, K.J.1
Jooss, K.2
Alston, J.3
-
41
-
-
80053399347
-
Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog
-
Koo T, Okada T, Athanasopoulos T, et al. Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog. J Gene Med 2011; 13: 497-506.
-
(2011)
J Gene Med
, vol.13
, pp. 497-506
-
-
Koo, T.1
Okada, T.2
Athanasopoulos, T.3
-
42
-
-
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
-
43
-
-
0029843596
-
Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector
-
Xiao X, Li J, Samulski RJ. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol 1996; 70: 8098-8108.
-
(1996)
J Virol
, vol.70
, pp. 8098-8108
-
-
Xiao, X.1
Li, J.2
Samulski, R.J.3
-
44
-
-
0029843596
-
Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector
-
Xiao X, Li J, Samulski RJ. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol 1996; 70: 8098-8108.
-
(1996)
J Virol
, vol.70
, pp. 8098-8108
-
-
Xiao, X.1
Li, J.2
Samulski, R.J.3
-
45
-
-
19344366672
-
Systemic protein delivery by muscle-gene transfer is limited by a local immune response
-
Wang L, Dobrzynski E, Schlachterman A, Cao O, Herzog RW. Systemic protein delivery by muscle-gene transfer is limited by a local immune response. Blood 2005; 105: 4226-4234.
-
(2005)
Blood
, vol.105
, pp. 4226-4234
-
-
Wang, L.1
Dobrzynski, E.2
Schlachterman, A.3
Cao, O.4
Herzog, R.W.5
-
46
-
-
0032977833
-
Induction of immunity to antigens expressed by recombinant adeno-associated virus depends on the route of administration
-
Brockstedt DG, Podsakoff GM, Fong L, et al. Induction of immunity to antigens expressed by recombinant adeno-associated virus depends on the route of administration. Clin Immunol 1999; 92: 67-75.
-
(1999)
Clin Immunol
, vol.92
, pp. 67-75
-
-
Brockstedt, D.G.1
Podsakoff, G.M.2
Fong, L.3
-
47
-
-
0030819372
-
Genetic immunization with adeno-associated virus vectors expressing herpes simplex virus type 2 glycoproteins B and D
-
Manning WC, Paliard X, Zhou S, et al. Genetic immunization with adeno-associated virus vectors expressing herpes simplex virus type 2 glycoproteins B and D. J Virol 1997; 71: 7960-7962.
-
(1997)
J Virol
, vol.71
, pp. 7960-7962
-
-
Manning, W.C.1
Paliard, X.2
Zhou, S.3
-
48
-
-
0034749458
-
Successful interference with cellular immune responses to immunogenic proteins encoded by recombinant viral vectors
-
Sarukhan A, Camugli S, Gjata B, et al. Successful interference with cellular immune responses to immunogenic proteins encoded by recombinant viral vectors. J Virol 2001; 75: 269-277.
-
(2001)
J Virol
, vol.75
, pp. 269-277
-
-
Sarukhan, A.1
Camugli, S.2
Gjata, B.3
-
49
-
-
4143097968
-
Therapeutic levels of factor IX expression using a muscle-specific promoter and adeno-associated virus serotype 1 vector
-
Liu YL, Mingozzi F, Rodriguez-Colon SM, et al. Therapeutic levels of factor IX expression using a muscle-specific promoter and adeno-associated virus serotype 1 vector. Hum Gene Ther 2004; 15: 783-792.
-
(2004)
Hum Gene Ther
, vol.15
, pp. 783-792
-
-
Liu, Y.L.1
Mingozzi, F.2
Rodriguez-Colon, S.M.3
-
50
-
-
0035915778
-
Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies
-
Cordier L, Gao GP, Hack AA, et al. Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies. Hum Gene Ther 2001; 12: 205-215.
-
(2001)
Hum Gene Ther
, vol.12
, pp. 205-215
-
-
Cordier, L.1
Gao, G.P.2
Hack, A.A.3
-
51
-
-
0035667634
-
Immune evasion by muscle-specific gene expression in dystrophic muscle
-
Hartigan-O'Connor D, Kirk CJ, Crawford R, Mule JJ, Chamberlain JS. Immune evasion by muscle-specific gene expression in dystrophic muscle. Mol Ther 2001; 4: 525-533.
-
(2001)
Mol Ther
, vol.4
, pp. 525-533
-
-
Hartigan-O'Connor, D.1
Kirk, C.J.2
Crawford, R.3
Mule, J.J.4
Chamberlain, J.S.5
-
52
-
-
3042767202
-
MicroRNAs: small RNAs with a big role in gene regulation
-
He L, Hannon GJ. MicroRNAs: small RNAs with a big role in gene regulation. Nat Rev Genet 2004; 5: 522-531.
-
(2004)
Nat Rev Genet
, vol.5
, pp. 522-531
-
-
He, L.1
Hannon, G.J.2
-
53
-
-
0037197803
-
Identification of tissue-specific microRNAs from mouse
-
Lagos-Quintana M, Rauhut R, Yalcin A, et al. Identification of tissue-specific microRNAs from mouse. Curr Biol 2002; 12: 735-739.
-
(2002)
Curr Biol
, vol.12
, pp. 735-739
-
-
Lagos-Quintana, M.1
Rauhut, R.2
Yalcin, A.3
-
54
-
-
36849053006
-
A microRNA-regulated lentiviral vector mediates stable correction of haemophilia B mice
-
Brown BD, Cantore A, Annoni A, et al. A microRNA-regulated lentiviral vector mediates stable correction of haemophilia B mice. Blood 2007; 110: 4144-4152.
-
(2007)
Blood
, vol.110
, pp. 4144-4152
-
-
Brown, B.D.1
Cantore, A.2
Annoni, A.3
-
55
-
-
36849060584
-
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state
-
Brown BD, Gentner B, Cantore A, et al. Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nat Biotechnol 2007; 25: 1457-1467.
-
(2007)
Nat Biotechnol
, vol.25
, pp. 1457-1467
-
-
Brown, B.D.1
Gentner, B.2
Cantore, A.3
-
56
-
-
84858124123
-
Impact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transduction
-
Cotugno G, Annunziata P, Barone MV, et al. Impact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transduction. PLoS One 2012; 7: e33286.
-
(2012)
PLoS One
, vol.7
-
-
Cotugno, G.1
Annunziata, P.2
Barone, M.V.3
-
57
-
-
79953803239
-
Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver
-
Qiao C, Yuan Z, Li J, et al. Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver. Gene Ther 2011; 18: 403-410.
-
(2011)
Gene Ther
, vol.18
, pp. 403-410
-
-
Qiao, C.1
Yuan, Z.2
Li, J.3
-
58
-
-
84894893260
-
Impact of the Host Environment on the Control of Gene Transfer-Induced Immune Responses with miRNA 142.3p-Regulated Vectors
-
Boisgerault F, Gross D, Ferrand M, et al. Impact of the Host Environment on the Control of Gene Transfer-Induced Immune Responses with miRNA 142.3p-Regulated Vectors. Mol Ther 2010; 18: S1-S20.
-
(2010)
Mol Ther
, vol.18
-
-
Boisgerault, F.1
Gross, D.2
Ferrand, M.3
|