Interventions for muscular dystrophy: molecular medicines entering the clinic

被引：26

作者：

Bushby, Kate ^{[1
]}

Lochmueller, Hanns ^{[1
]}

Lynn, Stephen ^{[1
]}

Straub, Volker ^{[1
]}

机构：

[1] Univ Newcastle, Inst Human Genet, Int Ctr Life, Newcastle Upon Tyne NE1 3BZ, Tyne & Wear, England

来源：

LANCET | 2009年 / 374卷 / 9704期

关键词：

SARCOGLYCAN-DEFICIENT MICE; RECOMBINANT ADENOASSOCIATED VIRUS; GENE-THERAPY; MOUSE MODEL; MUSCLE APOPTOSIS; SKELETAL-MUSCLE; MESSENGER-RNA; DYSTROGLYCAN GLYCOSYLATION; AUTOLOGOUS TRANSPLANTATION; NONSENSE MUTATIONS;

D O I：

10.1016/S0140-6736(09)61834-1

中图分类号：

R5 [内科学];

学科分类号：

100201 [内科学];

摘要：

Muscular dystrophies are individually rare genetic disorders that cause much chronic disability, affecting young children and adults. In the past 20 years, more than 30 genetic types of muscular dystrophy have been defined. During this time, precise diagnosis, genetic counselling, and medical management have improved. These advances in medical practice have occurred while definitive therapies based on an improved knowledge of disease pathogenesis are awaited. A wide range of therapeutic options have been tested in animal models, and some are being tested in clinical trials. Various therapeutic targets are being investigated, from personalised. medicines targeting specific mutations and drugs targeting cellular pathways to gene-based and cell-based therapies.

引用

收藏

页码：1849 / 1856

页数：8

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