Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

被引：734

作者：

Mingozzi, Federico ^{[1
]}

High, Katherine A. ^{[1
,2
,3
]}

机构：

[1] Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA

[2] Childrens Hosp Philadelphia, Howard Hughes Med Inst, Philadelphia, PA 19104 USA

[3] Univ Penn, Sch Med, Philadelphia, PA 19104 USA

来源：

NATURE REVIEWS GENETICS | 2011年 / 12卷 / 05期

基金：

美国国家卫生研究院;

关键词：

ADENOASSOCIATED VIRUS TYPE-2; CANINE HEMOPHILIA-B; HUMAN-FACTOR-IX; LONG-TERM CORRECTION; SUSTAINED PHENOTYPIC CORRECTION; LEBER CONGENITAL AMAUROSIS; LYSOSOMAL STORAGE DISEASE; DEFICIENT MOUSE MODEL; PHASE-I; SKELETAL-MUSCLE;

D O I：

10.1038/nrg2988

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

In vivo gene replacement for the treatment of inherited disease is one of the most compelling concepts in modern medicine. Adeno-associated virus (AAV) vectors have been extensively used for this purpose and have shown therapeutic efficacy in a range of animal models. Successful translation to the clinic was initially slow, but long-term expression of donated genes at therapeutic levels has now been achieved in patients with inherited retinal disorders and haemophilia B. Recent exciting results have raised hopes for the treatment of many other diseases. As we discuss here, the prospects and challenges for AAV gene therapy are to a large extent dependent on the target tissue and the specific disease.

引用

页码：341 / 355

页数：15

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