State-of-the-art gene-based therapies: the road ahead

被引：528

作者：

Kay, Mark A. ^{[1
]}

机构：

[1] Stanford Univ, Dept Pediat & Genet, Associate Pediat Chair Basic Res, Stanford, CA 94305 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; TRANSGENE EXPRESSION; FACTOR-IX; VECTOR INTEGRATION; HEMOPHILIA-B; EPIDERMOLYSIS-BULLOSA; ENDOGENOUS MICRORNA; SKELETAL-MUSCLE; CANINE MODEL; AAV VECTORS;

D O I：

10.1038/nrg2971

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

Improvements in the gene transfer vectors used in therapeutic trials have led to substantial clinical successes in patients with serious genetic conditions, such as immunodeficiency syndromes, blindness and some cancer types. Several barriers need to be overcome before this type of therapy becomes a widely accepted treatment for a broad group of medical diseases. However, recent progress in the field is finally realizing some of the promises made more than 20 years ago, providing optimism for additional successes in the near future.

引用

页码：316 / 328

页数：13

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