Gene therapy progress and prospects: gene therapy for severe combined immunodeficiency

被引：17

作者：

Gaspar, HB ^{[1
]}

Howe, S ^{[1
]}

Thrasher, AJ ^{[1
]}

机构：

[1] Inst Child Hlth, Mol Immunol Unit, London WC1N 1EH, England

来源：

GENE THERAPY | 2003年 / 10卷 / 24期

关键词：

SCID-X1; ADA; PEG-ADA; LMO-2; insertional mutagenesis;

D O I：

10.1038/sj.gt.3302150

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Severe combined immunodeficiencies have long been targeted as a group of disorders amenable to gene therapy because of their defined molecular biology and pathophysiology, and the prediction that corrected cells would have profound growth and survival advantage. Recently, several clinical studies have shown that conventional gene transfer technology can produce major beneficial therapeutic effects in these patients, but, as for all cellular and pharmacological treatment approaches, with a finite potential for toxicity.

引用

页码：1999 / 2004

页数：6

共 32 条

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