Strategies for Molecular Therapy of Duchenne Muscular Dystrophy
نویسندگان
چکیده
Expression of mini-dystrophin driven by the 1.35 kb MCK promoter ameliorates muscular dystrophy in fast, but not in slow muscles of transgenic mdx mice. Gentamicin fails to increase dystrophin expression in dystrophin-deficient muscle. (2003) U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping. Antibody-mediated targeting of an adenovirus vector modified to contain a synthetic immunoglobulin Ig-binding domain in the capsid. The short MCK1350 promoter/enhancer allows for sufficient dystrophin expression in skeletal muscles of mdx mice. Strategies for muscle-specific targeting of adenoviral gene transfer vectors.
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تاریخ انتشار 2003