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Volumn 27, Issue 4, 2016, Pages 143-149

Development of Optimized AAV Serotype Vectors for High-Efficiency Transduction at Further Reduced Doses

Author keywords

[No Author keywords available]

Indexed keywords

LYSINE; SERINE; THREONINE; TYROSINE; VIRUS VECTOR;

EID: 84983446228     PISSN: 19466536     EISSN: 19466544     Source Type: Journal    
DOI: 10.1089/hgtb.2016.054     Document Type: Article
Times cited : (17)

References (32)
  • 1
    • 44249120315 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.