-
1
-
-
70349481529
-
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1- AAT gene therapy
-
Brantly ML, et al. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1- AAT gene therapy. Proc Natl Acad Sci U S A. 2009;106(38):16363-16368.
-
(2009)
Proc Natl Acad Sci U S A
, vol.106
, Issue.38
, pp. 16363-16368
-
-
Brantly, M.L.1
-
2
-
-
79956000587
-
Gene therapy for alpha-1 antitrypsin deficiency
-
Flotte TR, Mueller C. Gene therapy for alpha-1 antitrypsin deficiency. Hum Mol Genet. 2011;20(R1):R87-R92.
-
(2011)
Hum Mol Genet
, vol.20
, Issue.R1
-
-
Flotte, T.R.1
Mueller, C.2
-
3
-
-
80155145248
-
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing alpha1-antitrypsin: Interim results
-
Flotte TR, et al. Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing alpha1-antitrypsin: interim results. Hum Gene Ther. 2011;22(10):1239-1247.
-
(2011)
Hum Gene Ther
, vol.22
, Issue.10
, pp. 1239-1247
-
-
Flotte, T.R.1
-
4
-
-
78249253608
-
Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D
-
Mendell JR, et al. Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Ann Neurol. 2010;68(5):629-638.
-
(2010)
Ann Neurol
, vol.68
, Issue.5
, pp. 629-638
-
-
Mendell, J.R.1
-
5
-
-
70350067897
-
Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins
-
Mendell JR, et al. Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Ann Neurol. 2009;66(3):290-297.
-
(2009)
Ann Neurol
, vol.66
, Issue.3
, pp. 290-297
-
-
Mendell, J.R.1
-
6
-
-
77957725001
-
Dystrophin immunity in Duchenne's muscular dystrophy
-
Mendell JR, et al. Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med. 2010;363(15):1429-1437.
-
(2010)
N Engl J Med
, vol.363
, Issue.15
, pp. 1429-1437
-
-
Mendell, J.R.1
-
7
-
-
84856520424
-
Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials
-
Wang Z, Tapscott SJ, Chamberlain JS, Storb R. Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials. Front Microbiol. 2011;2:201.
-
(2011)
Front Microbiol
, vol.2
, pp. 201
-
-
Wang, Z.1
Tapscott, S.J.2
Chamberlain, J.S.3
Storb, R.4
-
9
-
-
84859581254
-
Adenovirus type 35, but not type 5, stimulates NK cell activation via plasmacytoid dendritic cells and TLR9 signaling
-
Pahl JH, et al. Adenovirus type 35, but not type 5, stimulates NK cell activation via plasmacytoid dendritic cells and TLR9 signaling. Mol Immunol. 2012;51(1):91-100.
-
(2012)
Mol Immunol
, vol.51
, Issue.1
, pp. 91-100
-
-
Pahl, J.H.1
-
10
-
-
79952073161
-
Innate immune sensing of DNA viruses
-
Rathinam VA, Fitzgerald KA. Innate immune sensing of DNA viruses. Virology. 2011;411(2):153-162.
-
(2011)
Virology
, vol.411
, Issue.2
, pp. 153-162
-
-
Rathinam, V.A.1
Fitzgerald, K.A.2
-
11
-
-
32944464648
-
Pathogen recognition and innate immunity
-
DOI 10.1016/j.cell.2006.02.015, PII S0092867406001905
-
Akira S, Uematsu S, Takeuchi O. Pathogen recognition and innate immunity. Cell. 2006;124(4):783-801. (Pubitemid 43261452)
-
(2006)
Cell
, vol.124
, Issue.4
, pp. 783-801
-
-
Akira, S.1
Uematsu, S.2
Takeuchi, O.3
-
12
-
-
0034619794
-
A Toll-like receptor recognizes bacterial DNA
-
DOI 10.1038/35047123
-
Hemmi H, et al. A Toll-like receptor recognizes bacterial DNA. Nature. 2000;408(6813):740-745. (Pubitemid 32015992)
-
(2000)
Nature
, vol.408
, Issue.6813
, pp. 740-745
-
-
Hemmi, H.1
Takeuchi, O.2
Kawai, T.3
Kaisho, T.4
Sato, S.5
Sanjo, H.6
Matsumoto, M.7
Hoshino, K.8
Wagner, H.9
Takeda, K.10
Akira, S.11
-
13
-
-
0035137604
-
CpG motifs of DNA vaccines induce the expression of chemokines and MHC class II molecules on myocytes
-
Stan AC, Casares S, Brumeanu TD, Klinman DM, Bona CA. CpG motifs of DNA vaccines induce the expression of chemokines and MHC class II molecules on myocytes. Eur J Immunol. 2001;31(1):301-310.
-
(2001)
Eur J Immunol
, vol.31
, Issue.1
, pp. 301-310
-
-
Stan, A.C.1
Casares, S.2
Brumeanu, T.D.3
Klinman, D.M.4
Bona, C.A.5
-
14
-
-
0032400949
-
Human muscle cells express a functional costimulatory molecule distinct from B7.1 (CD80) and B7.2 (CD86) in vitro and in inflammatory lesions
-
Behrens L, Kerschensteiner M, Misgeld T, Goebels N, Wekerle H, Hohlfeld R. Human muscle cells express a functional costimulatory molecule distinct from B7.1 (CD80) and B7.2 (CD86) in vitro and in inflammatory lesions. J Immunol. 1998;161(11):5943-5951. (Pubitemid 28535962)
-
(1998)
Journal of Immunology
, vol.161
, Issue.11
, pp. 5943-5951
-
-
Behrens, L.1
Kerschensteiner, M.2
Misgeld, T.3
Goebels, N.4
Wekerle, H.5
Hohlfeld, R.6
-
15
-
-
79959201563
-
The genome of self- complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver
-
Martino AT, et al. The genome of self- complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver. Blood. 2011;117(24):6459-6468.
-
(2011)
Blood
, vol.117
, Issue.24
, pp. 6459-6468
-
-
Martino, A.T.1
-
16
-
-
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(8):2388-2398.
-
(2009)
J Clin Invest
, vol.119
, Issue.8
, pp. 2388-2398
-
-
Zhu, J.1
Huang, X.2
Yang, Y.3
-
17
-
-
84857784870
-
Self-complementary AAVs induce more potent transgene product-specific immune responses compared to a single-stranded genome
-
Wu T, et al. Self-complementary AAVs induce more potent transgene product-specific immune responses compared to a single-stranded genome. Mol Ther. 2012;20(3):572-579.
-
(2012)
Mol Ther
, vol.20
, Issue.3
, pp. 572-579
-
-
Wu, T.1
-
18
-
-
70350101102
-
+ T cell response to vector encoded proteins
-
+ T cell response to vector encoded proteins. J Immunol. 2009;182(10):6051-6060.
-
(2009)
J Immunol
, vol.182
, Issue.10
, pp. 6051-6060
-
-
Mays, L.E.1
-
19
-
-
0036227449
-
Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors
-
DOI 10.1128/JVI.76.9.4580-4590.2002
-
Zaiss AK, Liu Q, Bowen GP, Wong NC, Bartlett JS, Muruve DA. Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors. J Virol. 2002;76(9):4580-4590. (Pubitemid 34309593)
-
(2002)
Journal of Virology
, vol.76
, Issue.9
, pp. 4580-4590
-
-
Zaiss, A.-K.1
Liu, Q.2
Bowen, G.P.3
Wong, N.C.W.4
Bartlett, J.S.5
Muruve, D.A.6
-
20
-
-
1342311435
-
Contribution of Toll-like receptor 9 signaling to the acute inflammatory response to nonviral vectors
-
DOI 10.1016/j.ymthe.2003.11.012
-
Zhao H, Hemmi H, Akira S, Cheng SH, Scheule RK, Yew NS. Contribution of Toll-like receptor 9 signaling to the acute inflammatory response to nonviral vectors. Mol Ther. 2004;9(2):241-248. (Pubitemid 38256550)
-
(2004)
Molecular Therapy
, vol.9
, Issue.2
, pp. 241-248
-
-
Zhao, H.1
Hemmi, H.2
Akira, S.3
Cheng, S.H.4
Scheule, R.K.5
Yew, N.S.6
-
21
-
-
0034578390
-
Priming of CTL responses by DNA vaccines: Direct transfection of antigen presenting cells versus cross-priming
-
Ulmer JB, Otten GR. Priming of CTL responses by DNA vaccines: direct transfection of antigen presenting cells versus cross-priming. Dev Biol (Basel). 2000;104:9-14.
-
(2000)
Dev Biol (Basel)
, vol.104
, pp. 9-14
-
-
Ulmer, J.B.1
Otten, G.R.2
-
22
-
-
0030820743
-
Priming of cytotoxic T lymphocytes by DNA vaccines: Requirement for professional antigen presenting cells and evidence for antigen transfer from myocytes
-
Fu TM, et al. Priming of cytotoxic T lymphocytes by DNA vaccines: requirement for professional antigen presenting cells and evidence for antigen transfer from myocytes. Mol Med. 1997;3(6):362-371.
-
(1997)
Mol Med
, vol.3
, Issue.6
, pp. 362-371
-
-
Fu, T.M.1
-
23
-
-
80052017320
-
Evaluation of adeno- associated viral vectors for liver-directed gene transfer in dogs
-
Bell P, et al. Evaluation of adeno- associated viral vectors for liver-directed gene transfer in dogs. Hum Gene Ther. 2011;22(8):985-997.
-
(2011)
Hum Gene Ther
, vol.22
, Issue.8
, pp. 985-997
-
-
Bell, P.1
-
24
-
-
70349110562
-
Adeno-associated virus- mediated gene transfer to nonhuman primate liver can elicit destructive transgene- specific T cell responses
-
Gao G, et al. Adeno-associated virus- mediated gene transfer to nonhuman primate liver can elicit destructive transgene- specific T cell responses. Hum Gene Ther. 2009;20(9):930-942.
-
(2009)
Hum Gene Ther
, vol.20
, Issue.9
, pp. 930-942
-
-
Gao, G.1
-
25
-
-
74149088098
-
The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques
-
Wang L, et al. The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques. Mol Ther. 2010;18(1):126-134.
-
(2010)
Mol Ther
, vol.18
, Issue.1
, pp. 126-134
-
-
Wang, L.1
-
26
-
-
0033616680
-
Sustained correction of bleeding disorder in hemophilia B mice by gene therapy
-
DOI 10.1073/pnas.96.7.3906
-
Wang L, Takabe K, Bidlingmaier SM, Ill CR, Verma IM. Sustained correction of bleeding disorder in hemophilia B mice by gene therapy. Proc Natl Acad Sci U S A. 1999;96(7):3906-3910. (Pubitemid 29169009)
-
(1999)
Proceedings of the National Academy of Sciences of the United States of America
, vol.96
, Issue.7
, pp. 3906-3910
-
-
Wang, L.1
Takabe, K.2
Bidlingmaier, S.M.3
Ill, C.R.4
Verma, I.M.5
-
27
-
-
77958104835
-
Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale
-
Lock M, et al. Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Hum Gene Ther. 2010;21(10):1259-1271.
-
(2010)
Hum Gene Ther
, vol.21
, Issue.10
, pp. 1259-1271
-
-
Lock, M.1
|