AAV-Mediated Gene Therapy for Research and Therapeutic Purposes

被引：366
|
作者：
Samulski, R. Jude [1 ]
Muzyczka, Nicholas [2 ]
机构：
[1] Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27599 USA
[2] Univ Florida, Coll Med, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA
来源：
ANNUAL REVIEW OF VIROLOGY, VOL 1 | 2014年 / 1卷
关键词：
viral vector; DNA transfer; parvovirus; gene therapy; transduction; RECOMBINANT ADENOASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS; HERPES-SIMPLEX-VIRUS; HIGH-EFFICIENCY TRANSDUCTION; VIRAL VECTOR PRODUCTION; THYMIDINE KINASE GENE; DNA-BINDING PROTEIN; EXPRESSION IN-VIVO; TRANSGENE EXPRESSION; MUTATIONAL ANALYSIS;
D O I：
10.1146/annurev-virology-031413-085355
中图分类号：
Q93 [微生物学];
学科分类号：
071005 ; 100705 ;
摘要：
Adeno-associated virus (AAV) is a small, nonenveloped virus that was adapted 30 years ago for use as a gene transfer vehicle. It is capable of transducing a wide range of species and tissues in vivo with no evidence of toxicity, and it generates relatively mild innate and adaptive immune responses. We review the basic biology of AAV, the history of progress in AAV vector technology, and some of the clinical and research applications where AAV has shown success.
引用
收藏
页码：427 / 451
页数：25
相关论文
共 50 条
  • [1] AAV-Mediated Gene Therapy for Atherosclerosis
    Michael Lehrke
    Corinna Lebherz
    Current Atherosclerosis Reports, 2014, 16
  • [2] AAV-Mediated Gene Therapy for Atherosclerosis
    Lehrke, Michael
    Lebherz, Corinna
    CURRENT ATHEROSCLEROSIS REPORTS, 2014, 16 (09)
  • [3] AAV-mediated gene therapy for fALS
    Cappella, M.
    Cohen-Tannoudji, M.
    Marais, T.
    Astord, S.
    Besse, A.
    Giroux, B.
    Barkats, M.
    Biferi, M. G.
    HUMAN GENE THERAPY, 2018, 29 (12) : A109 - A109
  • [4] AAV-mediated gene therapy for sialidosis
    van de Vlekkert, Diantha
    Hu, Huimin
    Weesner, Jason A.
    Fremuth, Leigh E.
    Brown, Scott A.
    Lu, Meifen
    Gomero, Elida
    Campos, Yvan
    Sheppard, Heather
    d'Azzo, Alessandra
    MOLECULAR THERAPY, 2024, 32 (07) : 2094 - 2112
  • [5] AAV-mediated gene therapy for hemophilia
    Couto, LB
    Pierce, GF
    CURRENT OPINION IN MOLECULAR THERAPEUTICS, 2003, 5 (05) : 517 - 523
  • [6] AAV-mediated gene therapy of Pompe disease
    Ziegler, RJ
    Bercury, S
    Armentaro, D
    Raben, N
    Cheng, SH
    NEUROMUSCULAR DISORDERS, 2005, 15 (9-10) : 713 - 713
  • [7] AAV-Mediated Gene Therapy for the Treatment of Retinal Diseases
    Rolling, Fabienne
    CURRENT GENE THERAPY, 2010, 10 (05) : 318 - U4
  • [8] Prospects of AAV-mediated gene therapy for neuromuscular disease
    Voit, T.
    NEUROMUSCULAR DISORDERS, 2010, 20 (9-10) : 638 - 638
  • [9] Advancements in AAV-mediated Gene Therapy for Pompe Disease
    Salabarria, S. M.
    Nair, J.
    Clement, N.
    Smith, B. K.
    Raben, N.
    Fuller, D. D.
    Byrne, B. J.
    Corti, M.
    JOURNAL OF NEUROMUSCULAR DISEASES, 2020, 7 (01) : 15 - 31
  • [10] AAV-mediated gene therapy in a mouse model of sialidosis
    van de Vlekkert, Diantha
    Hu, Huimin
    Weesner, Jason A.
    Gomero, Elida M.
    d'Azzo, Alessandra
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 129 - 130
12345