Effective restoration of dystrophin-associated proteins in vivo by adenovirus-mediated transfer of truncated dystrophin cDNAs

被引:66
作者
Yuasa, K
Miyagoe, Y
Yamamoto, K
Nabeshima, Y
Dickson, G
Takeda, S
机构
[1] Natl Ctr Neurol & Psychiat, Natl Inst Neurosci, Dept Mol Genet, Tokyo 187, Japan
[2] Osaka Univ, Inst Mol & Cellular Biol, Osaka 565, Japan
[3] Univ London, Royal Holloway Coll, Sch Biol Sci, Surrey TW20 0EX, England
关键词
dystrophin; Duchenne muscular dystrophy; gene therapy; adenovirus vector; rod domain;
D O I
10.1016/S0014-5793(98)00251-8
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
A series of truncated dystrophin cDNAs (3.1-4.2 kbp) containing only three, three, two or one rod repeats with hinge 1 and 4 (named Delta DysAX2, AX11, AH3, M3, respectively) or no rod repeat retaining either hinge 1 or 4 (named Delta DysH1, H4, respectively) were constructed, These cDNAs were introduced into skeletal muscle of adult mdx mice using the adenovirus vector with a strong CAG promoter. Delta DysAX2, AX11, AH3 and Delta DysM3 expressed themselves successfully and recovered dystrophin-associated proteins effectively. Especially 3.7 kbp cDNA for Delta DysM3 offers the possibility of an approach utilizing newly developed virus vectors, such as an adeno-associated virus vector, toward gene therapy of Duchenne muscular dystrophy. (C) 1998 Federation of European Biochemical Societies.
引用
收藏
页码:329 / 336
页数:8
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