Engineering adeno-associated viruses for clinical gene therapy

被引:585
作者
Kotterman, Melissa A. [1 ]
Schaffer, David V. [1 ,2 ,3 ]
机构
[1] Univ Calif Berkeley, Dept Biomol & Chem Engn, Berkeley, CA 94720 USA
[2] Univ Calif Berkeley, Dept Bioengn, Berkeley, CA 94720 USA
[3] Univ Calif Berkeley, Dept Mol & Cell Biol, Berkeley, CA 94720 USA
基金
美国国家卫生研究院;
关键词
LEBERS CONGENITAL AMAUROSIS; LIPASE-DEFICIENT PATIENTS; CALCIUM UP-REGULATION; DIRECTED EVOLUTION; NEUTRALIZING ANTIBODIES; RPE65; MUTATIONS; NEXT-GENERATION; VIRAL VARIANT; AAV VECTOR; STEM-CELLS;
D O I
10.1038/nrg3742
中图分类号
Q3 [遗传学];
学科分类号
071007 ; 090102 ;
摘要
Clinical gene therapy has been increasingly successful owing both to an enhanced molecular understanding of human disease and to progressively improving gene delivery technologies. Among these technologies, delivery vectors based on adeno-associated viruses (AAVs) have emerged as safe and effective and, in one recent case, have led to regulatory approval. Although shortcomings in viral vector properties will render extension of such successes to many other human diseases challenging, new approaches to engineer and improve AAV vectors and their genetic cargo are increasingly helping to overcome these barriers.
引用
收藏
页码:445 / 451
页数:7
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