Gene therapy for lysosomal storage diseases

被引：195

作者：

Sands, Mark S.

Davidson, Beverly L.

机构：

[1] Washington Univ, Sch Med, Dept Internal Med, St Louis, MO 63110 USA

[2] Univ Iowa, Coll Med, Dept Internal Med, Program Gene Therapy, Iowa City, IA 52242 USA

[3] Univ Iowa, Coll Med, Dept Neurol, Iowa City, IA 52242 USA

[4] Univ Iowa, Coll Med, Dept Physiol & Biophys, Iowa City, IA 52242 USA

[5] Washington Univ, Sch Med, Dept Genet, St Louis, MO 63110 USA

来源：

MOLECULAR THERAPY | 2006年 / 13卷 / 05期

关键词：

D O I：

10.1016/j.ymthe.2006.01.006

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

Lysosomal storage diseases (LSDs) comprise a diverse group of monogenetic disorders with complex clinical phenotypes that include both systemic and central nervous system pathologies. In recent years, the identification or development of mouse models recapitulating the clinical course of the LSDs has been instrumental in evaluating therapeutic strategies. Here, we review the various gene replacement strategies for target organs affected in many LSDs and describe briefly the various vector systems employed to test how best to accomplish long-lasting therapies for these fatal disorders.

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页码：839 / 849

页数：11

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