EXPRESSION OF FULL-LENGTH AND TRUNCATED DYSTROPHIN MINI-GENES IN TRANSGENIC MDX MICE

被引：244

作者：

PHELPS, SF

HAUSER, MA

COLE, NM

RAFAEL, JA

HINKLE, RT

FAULKNER, JA

CHAMBERLAIN, JS

机构：

[1] UNIV MICHIGAN,SCH MED,DEPT HUMAN GENET,ANN ARBOR,MI 48109

[2] UNIV MICHIGAN,SCH MED,DEPT PHYSIOL,ANN ARBOR,MI 48109

[3] UNIV MICHIGAN,SCH MED,INST GERONTOL,ANN ARBOR,MI 48109

[4] UNIV MICHIGAN,SCH MED,CTR HUMAN GENOME,ANN ARBOR,MI 48109

来源：

HUMAN MOLECULAR GENETICS | 1995年 / 4卷 / 08期

关键词：

D O I：

10.1093/hmg/4.8.1251

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：

Duchenne and Becker muscular dystrophy are caused by defects in the dystrophin gene, and are candidates for treatment by gene therapy, We have shown previously that overexpression of a full-length dystrophin cDNA prevents the development of dystrophic symptoms in mdx mice, We show here that this functional correction can be achieved by expressing the full-length muscle isoform at a lower level than is present in control animals, Gene therapy for DMD may necessitate the use of truncated dystrophin mini-genes to accommodate the limited cloning capacity of current-generation viral delivery vectors, We have constructed both murine and human mini-genes deleted for exons 17-48, and have demonstrated that expression of either mini-gene can almost completely prevent the development of dystrophic symptoms in transgenic mdx mice, These results suggest that viral-mediated expression of moderate levels of a truncated dystrophin could be an effective treatment for DMD.

引用

页码：1251 / 1258

页数：8

共 95 条

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