-
1
-
-
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
-
2
-
-
44249085878
-
Safety and efficacy of gene transfer for Leber's congenital amaurosis
-
Maguire AM, Simonelli F, Pierce EA, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 2008;358:2240-2248.
-
(2008)
N Engl J Med
, vol.358
, pp. 2240-2248
-
-
Maguire, A.M.1
Simonelli, F.2
Pierce, E.A.3
-
3
-
-
54449085219
-
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
-
Cideciyan AV, Aleman TS, Boye SL, et al. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A 2008; 105:15112-15117.
-
(2008)
Proc Natl Acad Sci U S A
, vol.105
, pp. 15112-15117
-
-
Cideciyan, A.V.1
Aleman, T.S.2
Boye, S.L.3
-
4
-
-
54949104686
-
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: Short-term results of a phase i trial
-
Hauswirth WW, Aleman TS, Kaushal S, et al. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: Short-term results of a phase I trial. Hum Gene Ther 2008; 19:979-990.
-
(2008)
Hum Gene Ther
, vol.19
, pp. 979-990
-
-
Hauswirth, W.W.1
Aleman, T.S.2
Kaushal, S.3
-
5
-
-
84855161388
-
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
-
Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated 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
-
6
-
-
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
-
7
-
-
84861139370
-
Gene therapy for aromatic L-amino acid decarboxylase deficiency
-
Hwu WL, Muramatsu S, Tseng SH, et al. Gene therapy for aromatic L-amino acid decarboxylase deficiency. Sci Transl Med 2012;4:134ra61.
-
(2012)
Sci Transl Med
, vol.4
, pp. 134ra61
-
-
Hwu, W.L.1
Muramatsu, S.2
Tseng, S.H.3
-
8
-
-
84875962928
-
Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: An open-label trial
-
Gaudet D, Methot J, Dery S, et al. Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: An open-label trial. Gene Ther 2013; 20:361-369.
-
(2013)
Gene Ther
, vol.20
, pp. 361-369
-
-
Gaudet, D.1
Methot, J.2
Dery, S.3
-
9
-
-
84897051037
-
Retinal gene therapy in patients with choroideremia: Initial findings from a phase 1/2 clinical trial
-
MacLaren RE, Groppe M, Barnard AR, et al. Retinal gene therapy in patients with choroideremia: Initial findings from a phase 1/2 clinical trial. Lancet 2014;383:1129-1137.
-
(2014)
Lancet
, vol.383
, pp. 1129-1137
-
-
MacLaren, R.E.1
Groppe, M.2
Barnard, A.R.3
-
10
-
-
84883268937
-
Immune responses to AAV vectors: Overcoming barriers to successful gene therapy
-
Mingozzi F, High KA. Immune responses to AAV vectors: Overcoming barriers to successful gene therapy. Blood 2013;122:23-36.
-
(2013)
Blood
, vol.122
, pp. 23-36
-
-
Mingozzi, F.1
High, K.A.2
-
11
-
-
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
-
12
-
-
34147098413
-
CD8(+) T-cell responses to adeno-associated virus capsid in humans
-
Mingozzi F, Maus MV, Hui DJ, et al. CD8(+) T-cell 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
-
13
-
-
67651006589
-
Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors
-
Pien GC, Basner-Tschakarjan E, Hui DJ, et al. Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors. J Clin Invest 2009;119:1688-1695.
-
(2009)
J Clin Invest
, vol.119
, pp. 1688-1695
-
-
Pien, G.C.1
Basner-Tschakarjan, E.2
Hui, D.J.3
-
14
-
-
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
-
15
-
-
84875092627
-
Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: The final threshold?
-
Aslanidi GV, Rivers AE, Ortiz L, et al. Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: The final threshold? PLoS One 2013;8:e59142.
-
(2013)
PLoS One
, vol.8
, pp. e59142
-
-
Aslanidi, G.V.1
Rivers, A.E.2
Ortiz, L.3
-
16
-
-
84861189907
-
High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors
-
Aslanidi GV, Rivers AE, Ortiz L, et al. High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors. Vaccine 2012;30:3908-3917.
-
(2012)
Vaccine
, vol.30
, pp. 3908-3917
-
-
Aslanidi, G.V.1
Rivers, A.E.2
Ortiz, L.3
-
17
-
-
84920848456
-
Enhanced transgene expression from recombinant single-stranded dsequence-substituted adeno-associated virus vectors in human cell lines in vitro and in murine hepatocytes in vivo
-
Ling C, Wang Y, Lu Y, et al. Enhanced transgene expression from recombinant single-stranded Dsequence-substituted adeno-associated virus vectors in human cell lines in vitro and in murine hepatocytes in vivo. J Virol 2015;89:952-961.
-
(2015)
J Virol
, vol.89
, pp. 952-961
-
-
Ling, C.1
Wang, Y.2
Lu, Y.3
-
18
-
-
84944446155
-
Identification of small molecule inhibitors of zcchc11 TUTase activity
-
Lin S, Gregory RI. Identification of small molecule inhibitors of Zcchc11 TUTase activity. RNA Biol 2015;12:792-800.
-
(2015)
RNA Biol
, vol.12
, pp. 792-800
-
-
Lin, S.1
Gregory, R.I.2
-
19
-
-
84918780947
-
Cytotoxic genes from traditional Chinese medicine inhibit tumor growth both in vitro and in vivo
-
Zhang YH, Wang Y, Yusufali AH, et al. Cytotoxic genes from traditional Chinese medicine inhibit tumor growth both in vitro and in vivo. J Integr Med 2014;12:483-494.
-
(2014)
J Integr Med
, vol.12
, pp. 483-494
-
-
Zhang, Y.H.1
Wang, Y.2
Yusufali, A.H.3
-
20
-
-
84880550933
-
A concept of eliminating nonhomologous recombination for scalable and safe AAV vector generation for human gene therapy
-
Dong B, Moore AR, Dai J, et al. A concept of eliminating nonhomologous recombination for scalable and safe AAV vector generation for human gene therapy. Nucleic Acids Res 2013;41: 6609-6617.
-
(2013)
Nucleic Acids Res
, vol.41
, pp. 6609-6617
-
-
Dong, B.1
Moore, A.R.2
Dai, J.3
-
21
-
-
84906667355
-
Efficient production of dual recombinant adeno-associated viral vectors for factor VIII delivery
-
Wang Q, Dong B, Firrman J, et al. Efficient production of dual recombinant adeno-associated viral vectors for factor VIII delivery. Hum Gene Ther Methods 2014;25:261-268.
-
(2014)
Hum Gene Ther Methods
, vol.25
, pp. 261-268
-
-
Wang, Q.1
Dong, B.2
Firrman, J.3
-
22
-
-
85050580206
-
Prevalence of neutralizing antibodies against liver-tropic adenoassociated virus serotype vectors in 100 healthy Chinese and its potential relation to body constitutions
-
Ling C, Wang Y, Feng YL, et al. Prevalence of neutralizing antibodies against liver-tropic adenoassociated virus serotype vectors in 100 healthy Chinese and its potential relation to body constitutions. J Integr Med 2015;13:341-346.
-
(2015)
J Integr Med
, vol.13
, pp. 341-346
-
-
Ling, C.1
Wang, Y.2
Feng, Y.L.3
-
23
-
-
84924245829
-
The overexpression and nuclear translocation of trx-1 during hypoxia confers on HepG2 cells resistance to DDP, and GL-V9 reverses the resistance by suppressing the trx-1/Ref-1 axis
-
Zhao L, Li W, Zhou Y, et al. The overexpression and nuclear translocation of Trx-1 during hypoxia confers on HepG2 cells resistance to DDP, and GL-V9 reverses the resistance by suppressing the Trx-1/Ref-1 axis. Free Radic Biol Med 2015; 82:29-41.
-
(2015)
Free Radic Biol Med
, vol.82
, pp. 29-41
-
-
Zhao, L.1
Li, W.2
Zhou, Y.3
-
24
-
-
84885172948
-
DDX5 is a positive regulator of oncogenic NOTCH1 signaling in T cell acute lymphoblastic leukemia
-
Lin S, Tian L, Shen H, et al. DDX5 is a positive regulator of oncogenic NOTCH1 signaling in T cell acute lymphoblastic leukemia. Oncogene 2013;32: 4845-4853.
-
(2013)
Oncogene
, vol.32
, pp. 4845-4853
-
-
Lin, S.1
Tian, L.2
Shen, H.3
-
25
-
-
84918806026
-
Selective in vivo targeting of human liver tumors by optimized AAV3 vectors in a murine xenograft model
-
Ling C, Wang Y, Zhang Y, et al. Selective in vivo targeting of human liver tumors by optimized AAV3 vectors in a murine xenograft model. Hum Gene Ther 2014;25:1023-1034.
-
(2014)
Hum Gene Ther
, vol.25
, pp. 1023-1034
-
-
Ling, C.1
Wang, Y.2
Zhang, Y.3
-
26
-
-
84859610928
-
Development of optimized AAV3 serotype vectors: Mechanism of high-efficiency transduction of human liver cancer cells
-
Cheng B, Ling C, Dai Y, et al. Development of optimized AAV3 serotype vectors: Mechanism of high-efficiency transduction of human liver cancer cells. Gene Ther 2012;19:375-384.
-
(2012)
Gene Ther
, vol.19
, pp. 375-384
-
-
Cheng, B.1
Ling, C.2
Dai, Y.3
-
27
-
-
84955240212
-
Adeno-associated virus: The naturally occurring virus versus the recombinant vector
-
Srivastava A. Adeno-associated virus: The naturally occurring virus versus the recombinant vector. Hum Gene Ther 2016;27:1-6.
-
(2016)
Hum Gene Ther
, vol.27
, pp. 1-6
-
-
Srivastava, A.1
-
28
-
-
0346777307
-
Adenoassociated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo
-
McCarty DM, Fu H, Monahan PE, et al. Adenoassociated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther 2003;10:2112-2118.
-
(2003)
Gene Ther
, vol.10
, pp. 2112-2118
-
-
McCarty, D.M.1
Fu, H.2
Monahan, P.E.3
-
29
-
-
0348038748
-
Rapid and highly efficient transduction by double-stranded adenoassociated virus vectors in vitro and in vivo
-
Wang Z, Ma HI, Li J, et al. Rapid and highly efficient transduction by double-stranded adenoassociated virus vectors in vitro and in vivo. Gene Ther 2003;10:2105-2111.
-
(2003)
Gene Ther
, vol.10
, pp. 2105-2111
-
-
Wang, Z.1
Ma, H.I.2
Li, J.3
-
30
-
-
78650898983
-
High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines
-
Markusic DM, Herzog RW, Aslanidi GV, et al. High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines. Mol Ther 2010;18:2048-2056.
-
(2010)
Mol Ther
, vol.18
, pp. 2048-2056
-
-
Markusic, D.M.1
Herzog, R.W.2
Aslanidi, G.V.3
-
31
-
-
84877930020
-
Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsidspecific CD8+ T cells
-
Martino AT, Basner-Tschakarjan E, Markusic DM, et al. Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsidspecific 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
-
32
-
-
84959453256
-
Gene therapy for leber hereditary optic neuropathy: Initial results
-
Feuer WJ, Schiffman JC, Davis JL, et al. Gene therapy for Leber hereditary optic neuropathy: Initial results. Ophthalmology 2016;123:558-570.
-
(2016)
Ophthalmology
, vol.123
, pp. 558-570
-
-
Feuer, W.J.1
Schiffman, J.C.2
Davis, J.L.3
|