AAV-Mediated Gene Therapy for Research and Therapeutic Purposes

被引：395

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

基金：

美国国家卫生研究院;

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

页码：427 / 451

页数：25

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