-
1
-
-
0029151404
-
Adeno-associated virus vectors for gene therapy
-
Flotte TR, Carter BJ. Adeno-associated virus vectors for gene therapy. Gene Ther 1995;2:357-362.
-
(1995)
Gene Ther
, vol.2
, pp. 357-362
-
-
Flotte, T.R.1
Carter, B.J.2
-
2
-
-
0031300389
-
Gene therapy for the hemophilias
-
Walter J, High KA. Gene therapy for the hemophilias. Adv Vet Med 1997;40:119-134.
-
(1997)
Adv Vet Med
, vol.40
, pp. 119-134
-
-
Walter, J.1
High, K.A.2
-
3
-
-
4143097968
-
Therapeutic levels of factor IX expression using a musclespecific promoter and adeno-associated virus serotype 1 vector
-
Liu YL,Mingozzi F, Rodriguéz-Colôn SM, et al. Therapeutic levels of factor IX expression using a musclespecific 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
Rodriguéz-Colôn, S.M.3
-
4
-
-
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
-
5
-
-
59649122961
-
Longterm correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy
-
Niemeyer GP, Herzog RW, Mount J, et al. Longterm correction of inhibitor-prone hemophilia 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
-
6
-
-
84958212770
-
Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII
-
Marcos-Contreras OA, Smith SM, Bellinger DA, et al. Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII. Blood 2016;127:565-571.
-
(2016)
Blood
, vol.127
, pp. 565-571
-
-
Marcos-Contreras, O.A.1
Smith, S.M.2
Bellinger, D.A.3
-
7
-
-
33744900338
-
Highlevel transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes
-
Gao GP, Lu Y, Sun X, Johnston J, et al. Highlevel transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes. J Virol 2006;80:6192-6194.
-
(2006)
J Virol
, vol.80
, pp. 6192-6194
-
-
Gao, G.P.1
Lu, Y.2
Sun, X.3
Johnston, J.4
-
8
-
-
80455174705
-
AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta)
-
Wang L, Bell P, Lin J, et al. AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta). Mol Ther 2011;19:2012-2020.
-
(2011)
Mol Ther
, vol.19
, pp. 2012-2020
-
-
Wang, L.1
Bell, P.2
Lin, J.3
-
9
-
-
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;12:342-347.
-
(2006)
Nat Med
, vol.12
, pp. 342-347
-
-
Manno, C.S.1
Pierce, G.F.2
Arruda, V.R.3
-
10
-
-
84991408036
-
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: A follow-on phase 1 trial
-
Bennett J, Wellman J, Marshall KA, et al. Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial. Lancet 2016;388:661-672.
-
(2016)
Lancet
, vol.388
, pp. 661-672
-
-
Bennett, J.1
Wellman, J.2
Marshall, K.A.3
-
11
-
-
84928599756
-
The status of RPE65 gene therapy trials: Safety and efficacy
-
Pierce EA, Bennett J. The status of RPE65 gene therapy trials: safety and efficacy. Cold Spring Harb Perspect Med 2015;5:a017285.
-
(2015)
Cold Spring Harb Perspect Med
, vol.5
, pp. a017285
-
-
Pierce, E.A.1
Bennett, J.2
-
12
-
-
0033917280
-
Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease
-
Leone P, Janson CG, Bilaniuk L, et al. Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease. Ann Neurol 2000; 48:27-38.
-
(2000)
Ann Neurol
, vol.48
, pp. 27-38
-
-
Leone, P.1
Janson, C.G.2
Bilaniuk, L.3
-
13
-
-
34250683023
-
Safety and tolerability of gene therapy with an adenoassociated virus (AAV) borne GAD gene for Parkinson's disease: An open label, Phase I trial
-
Kaplitt MG, Feigin A, Tang C, et al. Safety and tolerability of gene therapy with an adenoassociated virus (AAV) borne GAD gene for Parkinson's disease: an open label, Phase I trial. Lancet 2007;369:2097-2105.
-
(2007)
Lancet
, vol.369
, pp. 2097-2105
-
-
Kaplitt, M.G.1
Feigin, A.2
Tang, C.3
-
15
-
-
34147098413
-
CD8(+) Tcell responses to adeno-associated virus capsid in humans
-
Mingozzi F, Maus MV, Hui DJ, et al. CD8(+) Tcell responses to adeno-associated virus capsid in humans. Nat Med 2007;13:419-422.
-
(2007)
Nat Med
, vol.13
, pp. 419-422
-
-
Mingozzi, F.1
Maus, M.V.2
Hui, D.J.3
-
16
-
-
85006785146
-
Preliminary results of a Phase 1/2 trial of SPK-9001, a hyperactive FIX variant delivered by a novel capsid, demonstrate consistent factor IX activity levels at the lowest dose cohort. WHF 2016, Late-Breaking Abstract
-
George LA, Sullivan S, Teitel J, et al. Preliminary results of a Phase 1/2 trial of SPK-9001, a hyperactive FIX variant delivered by a novel capsid, demonstrate consistent factor IX activity levels at the lowest dose cohort. WHF 2016, Late-Breaking Abstract. Haemophilia 2016;22:151-152.
-
(2016)
Haemophilia
, vol.22
, pp. 151-152
-
-
George, L.A.1
Sullivan, S.2
Teitel, J.3
-
17
-
-
0016632122
-
Isolation and characterization of an adenovirus and isolation of its adenovirusassociated virus in cell culture from foals with respiratory tract disease
-
Dutta SK. Isolation and characterization of an adenovirus and isolation of its adenovirusassociated virus in cell culture from foals with respiratory tract disease. Am J Vet Res 1975; 36:247-250.
-
(1975)
Am J Vet Res
, vol.36
, pp. 247-250
-
-
Dutta, S.K.1
-
18
-
-
0026516130
-
Detection of adeno-associated virus type 2 in human peripheral blood cells
-
Grossman Z, Mendelson E, Brok-Simoni F, et al. Detection of adeno-associated virus type 2 in human peripheral blood cells. Gen Virol 1992; 73:961-966.
-
(1992)
Gen Virol
, vol.73
, pp. 961-966
-
-
Grossman, Z.1
Mendelson, E.2
Brok-Simoni, F.3
-
19
-
-
0032551597
-
Detection of infectious adeno-associated virus particles in human cervical biopsies
-
Walz CM, Anisi TR, Schlehofer JR, et al. Detection of infectious adeno-associated virus particles in human cervical biopsies. Virology 1998;247:97-105.
-
(1998)
Virology
, vol.247
, pp. 97-105
-
-
Walz, C.M.1
Anisi, T.R.2
Schlehofer, J.R.3
-
20
-
-
27744592993
-
Molecular characterization of adeno-associated viruses infecting children
-
Chen CL, Jensen RL, Schnepp BC, et al. Molecular characterization of adeno-associated viruses infecting children. J Virol 2005;79:14781-14792.
-
(2005)
J Virol
, vol.79
, pp. 14781-14792
-
-
Chen, C.L.1
Jensen, R.L.2
Schnepp, B.C.3
-
21
-
-
0037947557
-
Adenoassociated viruses undergo substantial evolution in primates during natural infections
-
Gao G, Alvira MR, Somanathan S, et al. Adenoassociated viruses undergo substantial evolution in primates during natural infections. Proc Natl Acad Sci U S A 2003;100:6081-6086.
-
(2003)
Proc Natl Acad Sci U S A
, vol.100
, pp. 6081-6086
-
-
Gao, G.1
Alvira, M.R.2
Somanathan, S.3
-
22
-
-
33747874648
-
Adeno-associated virus (AAV) capsid genes isolated from rat and mouse liver genomic DNA define two new AAV species distantly related to AAV-5
-
Lochrie MA, Tatsuno GP, Arbetman AE, et al. Adeno-associated virus (AAV) capsid genes isolated from rat and mouse liver genomic DNA define two new AAV species distantly related to AAV-5. Virology 2006;353:68-82.
-
(2006)
Virology
, vol.353
, pp. 68-82
-
-
Lochrie, M.A.1
Tatsuno, G.P.2
Arbetman, A.E.3
-
23
-
-
3543100253
-
Characterization of the mouse adeno-associated virus AAVS1 ortholog
-
Dutheil N, Yoon-Robarts M, Ward P, et al. Characterization of the mouse adeno-associated virus AAVS1 ortholog. J Virol 2004;78:8917-8921.
-
(2004)
J Virol
, vol.78
, pp. 8917-8921
-
-
Dutheil, N.1
Yoon-Robarts, M.2
Ward, P.3
-
24
-
-
84876512832
-
Effects of immunosuppression on circulating adeno-associated virus capsid-specific T cells in humans
-
Parzych EM, Li H, Yin X, et al. Effects of immunosuppression on circulating adeno-associated virus capsid-specific T cells in humans. Hum Gene Ther 2013;24:431-442.
-
(2013)
Hum Gene Ther
, vol.24
, pp. 431-442
-
-
Parzych, E.M.1
Li, H.2
Yin, X.3
-
25
-
-
85018530032
-
Phenotypic and functional characterisation of human anti-AAV CD8+ T cells using MHC class I tetramer-associated magnetic enrichment
-
Vandamme C, Devaux M, Jaulin N, et al. Phenotypic and functional characterisation of human anti-AAV CD8+ T cells using MHC class I tetramer-associated magnetic enrichment. ASCGT meeting, 2016, Session Immunological Aspects of Gene Therapy II: AAV Vectors 697.
-
ASCGT Meeting, 2016, Session Immunological Aspects of Gene Therapy II: AAV Vectors 697
-
-
Vandamme, C.1
Devaux, M.2
Jaulin, N.3
-
26
-
-
58849086758
-
Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses
-
Calcedo R, Vandenberghe LH, Gao G, et al. Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis 2009;199:381-390.
-
(2009)
J Infect Dis
, vol.199
, pp. 381-390
-
-
Calcedo, R.1
Vandenberghe, L.H.2
Gao, G.3
-
27
-
-
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, et al. 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 2010;21:704-712.
-
(2010)
Hum Gene Ther
, vol.21
, pp. 704-712
-
-
Boutin, S.1
Monteilhet, V.2
Veron, P.3
-
28
-
-
84942905508
-
Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas
-
Nault JC, Datta S, Imbeaud S, et al. Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas. Nat Genet 2015;47: 1187-1193.
-
(2015)
Nat Genet
, vol.47
, pp. 1187-1193
-
-
Nault, J.C.1
Datta, S.2
Imbeaud, S.3
-
29
-
-
84962744063
-
Recombinant AAV integration is not associated with hepatic genotoxicity in nonhuman primates and patients
-
Gil-Farina I, Fronza R, Kaeppel C, et al. Recombinant AAV integration is not associated with hepatic genotoxicity in nonhuman primates and patients. Mol Ther 2016;24:1100-1105.
-
(2016)
Mol Ther
, vol.24
, pp. 1100-1105
-
-
Gil-Farina, I.1
Fronza, R.2
Kaeppel, C.3
-
30
-
-
84964335825
-
Wild-type AAV insertions in hepatocellular carcinoma do not inform debate over genotoxicity risk of vectorized AAV
-
Nault JC, Mami I, La Bella T, et al. Wild-type AAV insertions in hepatocellular carcinoma do not inform debate over genotoxicity risk of vectorized AAV. Mol Ther 2016;24:660-661.
-
(2016)
Mol Ther
, vol.24
, pp. 660-661
-
-
Nault, J.C.1
Mami, I.2
La Bella, T.3
-
31
-
-
84964388816
-
Reply to "Wild-type AAV insertions in hepatocellular carcinoma do not inform debate over genotoxicity risk of vectorized AAV"
-
Schmidt M, Gil-Farina I, Büning H. Reply to "Wild-type AAV insertions in hepatocellular carcinoma do not inform debate over genotoxicity risk of vectorized AAV." Mol Ther 2016; 24:661-662.
-
(2016)
Mol Ther
, vol.24
, pp. 661-662
-
-
Schmidt, M.1
Gil-Farina, I.2
Büning, H.3
-
32
-
-
9244234931
-
In vivo model of adeno-associated virus vector persistence and rescue
-
Afione SA, Conrad CK, Kearns WG, et al. In vivo model of adeno-associated virus vector persistence and rescue. J Virol 1996;70:3235-3241.
-
(1996)
J Virol
, vol.70
, pp. 3235-3241
-
-
Afione, S.A.1
Conrad, C.K.2
Kearns, W.G.3
-
33
-
-
0029257496
-
The cryptic life style of adeno-associated virus
-
Berns KI, Linden RM. The cryptic life style of adeno-associated virus. Bioessays 1995;17:237-245.
-
(1995)
Bioessays
, vol.17
, pp. 237-245
-
-
Berns, K.I.1
Linden, R.M.2
-
34
-
-
0038054338
-
AAV serotype 2 vectors preferentially integrate into active genes in mice
-
Nakai H, Montini E, Fuess S, et al. AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat Genet 2003;34:297-302.
-
(2003)
Nat Genet
, vol.34
, pp. 297-302
-
-
Nakai, H.1
Montini, E.2
Fuess, S.3
-
35
-
-
4344566457
-
Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system
-
Burger C, Gorbatyuk OS, Velardo MJ, et al. Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system. Mol Ther 2004;10:302-317.
-
(2004)
Mol Ther
, vol.10
, pp. 302-317
-
-
Burger, C.1
Gorbatyuk, O.S.2
Velardo, M.J.3
-
36
-
-
84985993659
-
In vivo tissue-tropism of adenoassociated viral vectors
-
Srivastava A. In vivo tissue-tropism of adenoassociated viral vectors. Curr Opin Virol 2016; 21:75-80.
-
(2016)
Curr Opin Virol
, vol.21
, pp. 75-80
-
-
Srivastava, A.1
-
37
-
-
44349170706
-
Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection
-
Zincarelli C, Soltys S, Rengo G, et al. Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol Ther 2008;16:1073-1080.
-
(2008)
Mol Ther
, vol.16
, pp. 1073-1080
-
-
Zincarelli, C.1
Soltys, S.2
Rengo, G.3
-
38
-
-
84894242894
-
Selection and evaluation of clinically relevant AAV variants in a xenograft liver model
-
Lisowski L, Dane AP, Chu K, et al. Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature 2014;506:382-386.
-
(2014)
Nature
, vol.506
, pp. 382-386
-
-
Lisowski, L.1
Dane, A.P.2
Chu, K.3
-
39
-
-
84964319994
-
Superior in vivo transduction of human hepatocytes using engineered AAV3 capsid
-
Vercauteren K, Hoffman BE, Zolotukhin I, et al. Superior in vivo transduction of human hepatocytes using engineered AAV3 capsid. Mol Ther 2016;24:1042-1049.
-
(2016)
Mol Ther
, vol.24
, pp. 1042-1049
-
-
Vercauteren, K.1
Hoffman, B.E.2
Zolotukhin, I.3
-
40
-
-
44949131860
-
In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adenoassociated viruses
-
Grimm D, Lee JS, Wang L, et al. In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adenoassociated viruses. J Virol 2008;82:5887-5911.
-
(2008)
J Virol
, vol.82
, pp. 5887-5911
-
-
Grimm, D.1
Lee, J.S.2
Wang, L.3
-
41
-
-
45549090635
-
Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses
-
Zhong L, Li B, Mah CS, et al. Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc Natl Acad Sci U S A 2008;105:7827-7832.
-
(2008)
Proc Natl Acad Sci U S A
, vol.105
, pp. 7827-7832
-
-
Zhong, L.1
Li, B.2
Mah, C.S.3
-
42
-
-
84978151018
-
Tyrosine mutation in AAV9 capsid improves gene transfer to the mouse lung
-
Martini SV, Silva AL, Ferreira D, et al. Tyrosine mutation in AAV9 capsid improves gene transfer to the mouse lung. Cell Physiol Biochem 2016; 39:544-553.
-
(2016)
Cell Physiol Biochem
, vol.39
, pp. 544-553
-
-
Martini, S.V.1
Silva, A.L.2
Ferreira, D.3
-
43
-
-
84879683527
-
CpG-depleted adeno-associated virus vectors evade immune detection
-
Faust SM, Bell P, Cutler BJ, et al. CpG-depleted adeno-associated virus vectors evade immune detection. J Clin Invest 2013;123:2994-3001.
-
(2013)
J Clin Invest
, vol.123
, pp. 2994-3001
-
-
Faust, S.M.1
Bell, P.2
Cutler, B.J.3
-
44
-
-
0034882826
-
Selfcomplementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis
-
McCarty DM, Monahan PE, Samulski RJ. Selfcomplementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther 2001;8:1248-1254.
-
(2001)
Gene Ther
, vol.8
, pp. 1248-1254
-
-
McCarty, D.M.1
Monahan, P.E.2
Samulski, R.J.3
-
45
-
-
33645528206
-
Selfcomplementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver
-
Nathwani AC, Gray JT, Ng CY, et al. Selfcomplementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood 2006;10:2653-2661.
-
(2006)
Blood
, vol.10
, pp. 2653-2661
-
-
Nathwani, A.C.1
Gray, J.T.2
Ng, C.Y.3
-
46
-
-
68849103488
-
The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice
-
Zhu J, Huang X, Yang Y. The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice. J Clin Invest 2009;119:2388-2398.
-
(2009)
J Clin Invest
, vol.119
, pp. 2388-2398
-
-
Zhu, J.1
Huang, X.2
Yang, Y.3
-
47
-
-
84929026406
-
Unique roles of TLR9-and MyD88-dependent and-independent pathways in adaptive immune responses to AAV-mediated gene transfer
-
Rogers GL, Suzuki M, Zolotukhin I, et al. Unique roles of TLR9-and MyD88-dependent and-independent pathways in adaptive immune responses to AAV-mediated gene transfer. J Innate Immun 2015;7:302-314.
-
(2015)
J Innate Immun
, vol.7
, pp. 302-314
-
-
Rogers, G.L.1
Suzuki, M.2
Zolotukhin, I.3
-
49
-
-
84155178352
-
Tolllike receptor 2-mediated innate immune response in human nonparenchymal liver cells toward adeno-associated viral vectors
-
Hösel M, Broxtermann M, Janicki H, et al. Tolllike receptor 2-mediated innate immune response in human nonparenchymal liver cells toward adeno-associated viral vectors. Hepatology 2012;55:287-297.
-
(2012)
Hepatology
, vol.55
, pp. 287-297
-
-
Hösel, M.1
Broxtermann, M.2
Janicki, H.3
-
50
-
-
37549003977
-
Prolonged susceptibility to antibody-mediated neutralization for adeno-associated vectors targeted to the liver
-
Murphy SL, Li H, Zhou S, et al. Prolonged susceptibility to antibody-mediated neutralization for adeno-associated vectors targeted to the liver. Mol Ther 2008;16:138-145.
-
(2008)
Mol Ther
, vol.16
, pp. 138-145
-
-
Murphy, S.L.1
Li, H.2
Zhou, S.3
-
51
-
-
84880559842
-
Overcoming preexisting humoral immunity to AAV using capsid decoys
-
Mingozzi F, Anguela XM, Pavani G, et al. Overcoming preexisting humoral immunity to AAV using capsid decoys. Sci Transl Med 2013;5: 194ra92.
-
(2013)
Sci Transl Med
, vol.5
, pp. 194ra92
-
-
Mingozzi, F.1
Anguela, X.M.2
Pavani, G.3
-
52
-
-
28444491760
-
Identification of mouse AAV capsid-specific CD8+ T cell epitopes
-
Sabatino DE, Mingozzi F, Hui DJ, et al. Identification of mouse AAV capsid-specific CD8+ T cell epitopes. Mol Ther 2005;12:1023-1033.
-
(2005)
Mol Ther
, vol.12
, pp. 1023-1033
-
-
Sabatino, D.E.1
Mingozzi, F.2
Hui, D.J.3
-
53
-
-
84945932371
-
AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes
-
Hui DJ, Edmonson SC, Podsakoff GM, et al. AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes. Mol Ther Methods Clin Dev 2015;2:15029.
-
(2015)
Mol Ther Methods Clin Dev
, vol.2
, pp. 15029
-
-
Hui, D.J.1
Edmonson, S.C.2
Podsakoff, G.M.3
-
54
-
-
80455174048
-
Capsid-specific T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primates
-
Li H, Lasaro MO, Jia B, et al. Capsid-specific T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primates. Mol Ther 2011;19: 2021-2030.
-
(2011)
Mol Ther
, vol.19
, pp. 2021-2030
-
-
Li, H.1
Lasaro, M.O.2
Jia, B.3
-
55
-
-
70049118306
-
Isolation and characterization of adenoviruses persistently shed from the gastrointestinal tract of non-human primates
-
Roy S, Vandenberghe LH, Kryazhimskiy S, et al. Isolation and characterization of adenoviruses persistently shed from the gastrointestinal tract of non-human primates. PLoS Pathog 2009;5: e1000503.
-
(2009)
PLoS Pathog
, vol.5
, pp. e1000503
-
-
Roy, S.1
Vandenberghe, L.H.2
Kryazhimskiy, S.3
-
56
-
-
79956077563
-
T cell exhaustion
-
Wherry EJ. T cell exhaustion. Nat Immunol 2011; 12:492-499.
-
(2011)
Nat Immunol
, vol.12
, pp. 492-499
-
-
Wherry, E.J.1
-
57
-
-
84875443169
-
Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis
-
Sondhi D, Johnson L, Purpura K, et al. Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis. Hum Gene Ther Methods 2012;23:324-335.
-
(2012)
Hum Gene Ther Methods
, vol.23
, pp. 324-335
-
-
Sondhi, D.1
Johnson, L.2
Purpura, K.3
-
58
-
-
33744825168
-
Immune responses to AAV in a Phase I study for Canavan disease
-
McPhee SW, Janson CG, Li C, et al. Immune responses to AAV in a Phase I study for Canavan disease. J Gene Med 2006;8:577-588.
-
(2006)
J Gene Med
, vol.8
, pp. 577-588
-
-
McPhee, S.W.1
Janson, C.G.2
Li, C.3
-
59
-
-
44349136847
-
Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA
-
Worgall S, Sondhi D, Hackett NR, et al Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. Hum Gene Ther 2008;19:463-474.
-
(2008)
Hum Gene Ther
, vol.19
, pp. 463-474
-
-
Worgall, S.1
Sondhi, D.2
Hackett, N.R.3
-
60
-
-
84911383748
-
Long-term safety and efficacy of factor IX gene therapy in hemophilia B
-
Nathwani AC, Reiss UM, Tuddenham EG, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014; 371:1994-2004.
-
(2014)
N Engl J Med
, vol.371
, pp. 1994-2004
-
-
Nathwani, A.C.1
Reiss, U.M.2
Tuddenham, E.G.3
-
61
-
-
80155145248
-
Phase 2 clinical trial of a recombinant adenoassociated viral vector expressing a1-antitrypsin: Interim results
-
Flotte TR, Trapnell BC, Humphries M, et al. Phase 2 clinical trial of a recombinant adenoassociated viral vector expressing a1-antitrypsin: interim results. Hum Gene Ther 2011;22:1239-1247.
-
(2011)
Hum Gene Ther
, vol.22
, pp. 1239-1247
-
-
Flotte, T.R.1
Trapnell, B.C.2
Humphries, M.3
-
62
-
-
33947196295
-
Preexisting AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes
-
Li H, Murphy SL, Giles-Davis W, et al. Preexisting AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes. Mol Ther 2007;15:792-800.
-
(2007)
Mol Ther
, vol.15
, pp. 792-800
-
-
Li, H.1
Murphy, S.L.2
Giles-Davis, W.3
-
63
-
-
67649849935
-
A preclinical animal model to assess the effect of pre-existing immunity on AAV-mediated gene transfer
-
Li H, Lin SW, Giles-Davis W, et al. A preclinical animal model to assess the effect of pre-existing immunity on AAV-mediated gene transfer. Mol Ther 2009;17:1215-1224.
-
(2009)
Mol Ther
, vol.17
, pp. 1215-1224
-
-
Li, H.1
Lin, S.W.2
Giles-Davis, W.3
-
64
-
-
79952195654
-
Adenoassociated virus vectors serotype 2 induce prolonged proliferation of capsid-specific CD8+ T cells in mice
-
Li H, Tuyishime S, Wu TL, et al. Adenoassociated virus vectors serotype 2 induce prolonged proliferation of capsid-specific CD8+ T cells in mice. Mol Ther 2011;19:536-546.
-
(2011)
Mol Ther
, vol.19
, pp. 536-546
-
-
Li, H.1
Tuyishime, S.2
Wu, T.L.3
-
65
-
-
34447281279
-
Adenoassociated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vectortransduced cells coexpressing the AAV2 capsid in vivo
-
Li C, Hirsch M, Asokan A, et al. Adenoassociated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vectortransduced cells coexpressing the AAV2 capsid in vivo. J Virol 2007;81:7540-7547.
-
(2007)
J Virol
, vol.81
, pp. 7540-7547
-
-
Li, C.1
Hirsch, M.2
Asokan, A.3
-
66
-
-
84877930020
-
Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells
-
Martino AT, Basner-Tschakarjan E, Markusic DM, et al. Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells. Blood 2013;121: 2224-2233.
-
(2013)
Blood
, vol.121
, pp. 2224-2233
-
-
Martino, A.T.1
Basner-Tschakarjan, E.2
Markusic, D.M.3
-
67
-
-
33746801474
-
Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid
-
Vandenberghe LH, Wang L, Somanathan S, et al. Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat Med 2006;12:967-971.
-
(2006)
Nat Med
, vol.12
, pp. 967-971
-
-
Vandenberghe, L.H.1
Wang, L.2
Somanathan, S.3
-
68
-
-
58149251820
-
Undetectable transcription of cap in a clinical AAV vector: Implications for preformed capsid in immune responses
-
Hauck B, Murphy SL, Smith PH, et al. Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses. Mol Ther 2009;17:144-152.
-
(2009)
Mol Ther
, vol.17
, pp. 144-152
-
-
Hauck, B.1
Murphy, S.L.2
Smith, P.H.3
-
69
-
-
0030176270
-
Evidence that a single peptide-MHC complex on a target cell can elicit a cytolytic T cell response
-
Sykulev Y, Joo M, Vturina I, et al. Evidence that a single peptide-MHC complex on a target cell can elicit a cytolytic T cell response. Immunity 1996;4:565-571.
-
(1996)
Immunity
, vol.4
, pp. 565-571
-
-
Sykulev, Y.1
Joo, M.2
Vturina, I.3
-
70
-
-
84975516333
-
The comings and goings of MHC class I molecules herald a new dawn in crosspresentation
-
Blander JM. The comings and goings of MHC class I molecules herald a new dawn in crosspresentation. Immunol Rev 2016;272:65-79.
-
(2016)
Immunol Rev
, vol.272
, pp. 65-79
-
-
Blander, J.M.1
-
71
-
-
34247200575
-
Crosspresentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets
-
Wang L, Figueredo J, Calcedo R, et al. Crosspresentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets. Hum Gene Ther 2007;18:185-194.
-
(2007)
Hum Gene Ther
, vol.18
, pp. 185-194
-
-
Wang, L.1
Figueredo, J.2
Calcedo, R.3
-
72
-
-
84874594941
-
Adenoassociated virus capsid antigen presentation is dependent on endosomal escape
-
Li C, He Y, Nicolson S, Hirsch M, et al. Adenoassociated virus capsid antigen presentation is dependent on endosomal escape. J Clin Invest 2013;123:1390-1401.
-
(2013)
J Clin Invest
, vol.123
, pp. 1390-1401
-
-
Li, C.1
He, Y.2
Nicolson, S.3
Hirsch, M.4
-
73
-
-
67649817113
-
Cellular immune response to cryptic epitopes during therapeutic gene transfer
-
Li C, Goudy K, Hirsch M, et al. Cellular immune response to cryptic epitopes during therapeutic gene transfer. Proc Natl Acad Sci U S A 2009; 106:10770-10774.
-
(2009)
Proc Natl Acad Sci U S A
, vol.106
, pp. 10770-10774
-
-
Li, C.1
Goudy, K.2
Hirsch, M.3
-
74
-
-
44249120315
-
Effect of gene therapy on visual function in Leber's congenital amaurosis
-
Bainbridge JW, Smith AJ, Barker SS, et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 2008;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
-
75
-
-
70350620424
-
Agedependent effects of RPE65 gene therapy for Leber's congenital amaurosis: A Phase 1 doseescalation trial
-
Maguire AM, High KA, Auricchio A, et al. Agedependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a Phase 1 doseescalation trial. Lancet 2009;374:1597-1605.
-
(2009)
Lancet
, vol.374
, pp. 1597-1605
-
-
Maguire, A.M.1
High, K.A.2
Auricchio, A.3
-
76
-
-
84932084122
-
Proof of concept for AAV2/5-mediated gene therapy in iPSC-derived retinal pigment epithelium of a choroideremia patient
-
Cereso N, Pequignot MO, Robert L, et al. Proof of concept for AAV2/5-mediated gene therapy in iPSC-derived retinal pigment epithelium of a choroideremia patient. Mol Ther Methods Clin Dev 2014;1:14011.
-
(2014)
Mol Ther Methods Clin Dev
, vol.1
, pp. 14011
-
-
Cereso, N.1
Pequignot, M.O.2
Robert, L.3
-
77
-
-
0034724209
-
Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system
-
Davidson BL, Stein CS, Heth JA, et al. Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system. Proc Natl Acad Sci U S A 2000;97:3428-3432.
-
(2000)
Proc Natl Acad Sci U S A
, vol.97
, pp. 3428-3432
-
-
Davidson, B.L.1
Stein, C.S.2
Heth, J.A.3
-
78
-
-
44949222522
-
Results from a phase I safety trial of hAADC gene therapy for Parkinson disease
-
Eberling JL, Jagust WJ, Christine CW, et al. Results from a phase I safety trial of hAADC gene therapy for Parkinson disease. Neurology 2008;70:1980-1983.
-
(2008)
Neurology
, vol.70
, pp. 1980-1983
-
-
Eberling, J.L.1
Jagust, W.J.2
Christine, C.W.3
-
79
-
-
79952740079
-
AAV2-GAD gene therapy for advanced Parkinson's disease: A double-blind, sham-surgery controlled, randomised trial
-
LeWitt PA, Rezai AR, Leehey MA, et al. AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial. Lancet Neurol 2011; 10:309-319.
-
(2011)
Lancet Neurol
, vol.10
, pp. 309-319
-
-
LeWitt, P.A.1
Rezai, A.R.2
Leehey, M.A.3
-
80
-
-
70349481529
-
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
-
Brantly ML, Chulay JD, Wang L, et al. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc Natl Acad Sci USA 2009;106: 16363-16368.
-
(2009)
Proc Natl Acad Sci USA
, vol.106
, pp. 16363-16368
-
-
Brantly, M.L.1
Chulay, J.D.2
Wang, L.3
-
81
-
-
84890054617
-
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression
-
Mueller C, Chulay JD, Trapnell BC, et al. Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression. J Clin Invest. 2013;123:5310-5318.
-
(2013)
J Clin Invest.
, vol.123
, pp. 5310-5318
-
-
Mueller, C.1
Chulay, J.D.2
Trapnell, B.C.3
-
82
-
-
0021190074
-
The detailed distribution of HLA-A, B, C antigens in normal human organs
-
Daar AS, Fuggle SV, Fabre JW, et al. The detailed distribution of HLA-A, B, C antigens in normal human organs. Transplantation 1984;38: 287-292.
-
(1984)
Transplantation
, vol.38
, pp. 287-292
-
-
Daar, A.S.1
Fuggle, S.V.2
Fabre, J.W.3
-
83
-
-
0024789276
-
Interferon-gamma-like immunoreactivity and T-cell marker expression in rat skeletal muscle fibres
-
Nennesmo I, Olsson T, Ljungdahl A, et al. Interferon-gamma-like immunoreactivity and T-cell marker expression in rat skeletal muscle fibres. Brain Res 1989;504:306-310.
-
(1989)
Brain Res
, vol.504
, pp. 306-310
-
-
Nennesmo, I.1
Olsson, T.2
Ljungdahl, A.3
-
84
-
-
0242422840
-
Human muscle cells express a B7-related molecule, B7-H1, with strong negative immune regulatory potential: A novel mechanism of counterbalancing the immune attack in idiopathic inflammatory myopathies
-
Wiendl H, Mitsdoerffer M, Schneider D, et al. Human muscle cells express a B7-related molecule, B7-H1, with strong negative immune regulatory potential: a novel mechanism of counterbalancing the immune attack in idiopathic inflammatory myopathies. FASEB J 2003; 17:1892-1894.
-
(2003)
FASEB J
, vol.17
, pp. 1892-1894
-
-
Wiendl, H.1
Mitsdoerffer, M.2
Schneider, D.3
-
85
-
-
55849101706
-
Alpha1-antitrypsin monotherapy induces immune tolerance during islet allograft transplantation in mice
-
Lewis EC, Mizrahi M, Toledano M, et al. Alpha1-antitrypsin monotherapy induces immune tolerance during islet allograft transplantation in mice. Proc Natl Acad Sci U S A 2008;105:16236-16241.
-
(2008)
Proc Natl Acad Sci U S A
, vol.105
, pp. 16236-16241
-
-
Lewis, E.C.1
Mizrahi, M.2
Toledano, M.3
-
86
-
-
24644461309
-
Alpha1-antitrypsin monotherapy prolongs islet allograft survival in mice
-
Lewis EC, Shapiro L, Bowers OJ, et al. Alpha1-antitrypsin monotherapy prolongs islet allograft survival in mice. Proc Natl Acad Sci U S A 2005; 102:12153-12158.
-
(2005)
Proc Natl Acad Sci U S A
, vol.102
, pp. 12153-12158
-
-
Lewis, E.C.1
Shapiro, L.2
Bowers, O.J.3
-
87
-
-
84858776868
-
A-1-antitrypsin gene delivery reduces inflammation, increases T-regulatory cell population size and prevents islet allograft rejection
-
Shahaf G, Moser H, Ozeri E, et al. a-1-antitrypsin gene delivery reduces inflammation, increases T-regulatory cell population size and prevents islet allograft rejection. Mol Med 2011;171000-171011.
-
(2011)
Mol Med
, pp. 171000-171011
-
-
Shahaf, G.1
Moser, H.2
Ozeri, E.3
-
88
-
-
79959696178
-
Recent advances in cytometry applications: Preclinical, clinical, and cell biology
-
Mittag A, Tarnok A. Recent advances in cytometry applications: preclinical, clinical, and cell biology. Methods Cell Biol 2011;103:1-20.
-
(2011)
Methods Cell Biol
, vol.103
, pp. 1-20
-
-
Mittag, A.1
Tarnok, A.2
-
90
-
-
64049093048
-
Reducing the risk of adeno-associated virus (AAV) vector mobilization with AAV type 5 vectors
-
Hewitt FC, Li C, Gray SJ, et al. Reducing the risk of adeno-associated virus (AAV) vector mobilization with AAV type 5 vectors. J Virol 2009; 83:3919-3929.
-
(2009)
J Virol
, vol.83
, pp. 3919-3929
-
-
Hewitt, F.C.1
Li, C.2
Gray, S.J.3
-
91
-
-
33645923713
-
2006. Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2, 5, and 6 in cystic fibrosis and normal populations: Implications for gene therapy using AAV vectors
-
Halbert CL, Miller AD, Mcnamara S, et al. 2006. Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2, 5, and 6 in cystic fibrosis and normal populations: implications for gene therapy using AAV vectors. Hum Gene Ther 2006;17:440-447.
-
(2006)
Hum Gene Ther
, vol.17
, pp. 440-447
-
-
Halbert, C.L.1
Miller, A.D.2
Mcnamara, S.3
-
92
-
-
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
-
93
-
-
85006791028
-
Updated results from a dose escalating study in adult patients with haemophilia B treated with AMT-060 (AAV5-hFIX) gene therapy. WHF 2016, Late-Breaking Abstract
-
Miesbach W, Klamroth R, Schutgens R, et al. Updated results from a dose escalating study in adult patients with haemophilia B treated with AMT-060 (AAV5-hFIX) gene therapy. WHF 2016, Late-Breaking Abstract. Haemophilia 2016;22: 151-152.
-
(2016)
Haemophilia
, vol.22
, pp. 151-152
-
-
Miesbach, W.1
Klamroth, R.2
Schutgens, R.3
-
94
-
-
85011356650
-
Interim results of an open-label, Phase 1/2 study of BMN 270, an AAV5-FVIII gene transfer in severe hemophilia A, WHF 2016, Late-Breaking Abstract
-
Pasi J, Rangarajan S, Pierce G, et al. Interim results of an open-label, Phase 1/2 study of BMN 270, an AAV5-FVIII gene transfer in severe hemophilia A, WHF 2016, Late-Breaking Abstract. Haemophilia 2016;22:151-152.
-
(2016)
Haemophilia
, vol.22
, pp. 151-152
-
-
Pasi, J.1
Rangarajan, S.2
Pierce, G.3
-
95
-
-
84855161388
-
Adenovirus-associated virus vectormediated gene transfer in hemophilia B
-
Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vectormediated gene transfer in hemophilia 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
-
96
-
-
84896320924
-
Immune responses to intramuscular administration of alipogene tiparvovec (AAV1-LPL(S447X)) in a Phase II clinical trial of lipoprotein lipase deficiency gene therapy
-
Ferreira V, Twisk J, Kwikkers K, et al. Immune responses to intramuscular administration of alipogene tiparvovec (AAV1-LPL(S447X)) in a Phase II clinical trial of lipoprotein lipase deficiency gene therapy. Hum Gene Ther 2014;25:180-188.
-
(2014)
Hum Gene Ther
, vol.25
, pp. 180-188
-
-
Ferreira, V.1
Twisk, J.2
Kwikkers, K.3
-
97
-
-
84946558096
-
Update on a Phase 1/2 open-label trial of BAX335, an adeno-associated virus 8 (AAV8) vector-based gene therapy program for hemophilia B
-
June 24, Toronto, Canada, Abstract #LB101
-
Monahan P, Walsh CE, Powell JS, et al. Update on a Phase 1/2 open-label trial of BAX335, an adeno-associated virus 8 (AAV8) vector-based gene therapy program for hemophilia B. International Society on Thrombosis and Haemostasis 2015 Congress, June 24, 2015, Toronto, Canada, Abstract #LB101.
-
(2015)
International Society On Thrombosis and Haemostasis 2015 Congress
-
-
Monahan, P.1
Walsh, C.E.2
Powell, J.S.3
-
98
-
-
84924997217
-
AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice
-
Crudele JM, Finn JD, Siner JI, et al. AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice. Blood 2015;125: 1553-1561.
-
(2015)
Blood
, vol.125
, pp. 1553-1561
-
-
Crudele, J.M.1
Finn, J.D.2
Siner, J.I.3
-
99
-
-
70449103126
-
A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells
-
Smith RH, Levy JR, Kotin RM. A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells. Mol Ther 2009;17:1888-1896.
-
(2009)
Mol Ther
, vol.17
, pp. 1888-1896
-
-
Smith, R.H.1
Levy, J.R.2
Kotin, R.M.3
-
100
-
-
32444440088
-
Scalable generation of high-titer recombinant adenoassociated virus type 5 in insect cells
-
Urabe M, Nakakura T, Xin KQ, et al. Scalable generation of high-titer recombinant adenoassociated virus type 5 in insect cells. J Virol 2006;80:1874-1885.
-
(2006)
J Virol
, vol.80
, pp. 1874-1885
-
-
Urabe, M.1
Nakakura, T.2
Xin, K.Q.3
-
101
-
-
84876171439
-
Structure and dynamics of adeno-associated virus serotype 1 VP1-unique N-terminal domain and its role in capsid trafficking
-
Venkatakrishnan B, Yarbrough J, Domsic J, et al. Structure and dynamics of adeno-associated virus serotype 1 VP1-unique N-terminal domain and its role in capsid trafficking. J Virol 2013;87:4974-4984.
-
(2013)
J Virol
, vol.87
, pp. 4974-4984
-
-
Venkatakrishnan, B.1
Yarbrough, J.2
Domsic, J.3
-
102
-
-
84945560129
-
One-Bac 2.0: Sf9 cell lines for production of AAV5 vectors with enhanced infectivity and minimal encapsidation of foreign DNA
-
Mietzsch M, Casteleyn V, Weger S, et al. One-Bac 2.0: Sf9 cell lines for production of AAV5 vectors with enhanced infectivity and minimal encapsidation of foreign DNA. Hum Gene Ther 2015;26:688-697.
-
(2015)
Hum Gene Ther
, vol.26
, pp. 688-697
-
-
Mietzsch, M.1
Casteleyn, V.2
Weger, S.3
|