Electrotransfer of the Full-Length Dog Dystrophin into Mouse and Dystrophic Dog Muscles

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Electrotransfer of the Full-Length Dog Dystrophin into Mouse and Dystrophic Dog Muscles

Authors
  • 1 AIMS , (United States)
Type
Published Article
Journal
Human Gene Therapy
Publisher
Mary Ann Liebert
Volume
21
Issue
11
Pages
1591–1601
Identifiers
DOI: 10.1089/hum.2010.024
Source
AIMS
Keywords
License
Green

Abstract

Duchenne muscular dystrophy (DMD) is an X-linked genetic disease characterized by the absence of dystrophin (427 kDa). An approach to eventually restore this protein in patients with DMD is to introduce into their muscles a plasmid encoding dystrophin cDNA. Because the phenotype of the dystrophic dog is closer to the human phenotype than is the mdx mouse phenotype, we have studied the electrotransfer of a plasmid carrying the full-length dog dystrophin (FLDYS dog) in dystrophic dog muscle. To achieve this nonviral delivery, the FLDYS dog cDNA was cloned in two plasmids containing either a cytomegalovirus or a muscle creatine kinase promoter. In both cases, our results showed that the electrotransfer of these large plasmids (*17 kb) into mouse muscle allowed FLDYS dog expression in the treated muscle. The electrotransfer of pCMV.FLDYS dog in a dystrophic dog muscle also led to the expression of dystrophin. In conclusion, introduction of the full-length dog dystrophin cDNA by electrotransfer into dystrophic dog muscle is a potential approach to restore dystrophin in patients with DMD. However, the electrotransfer procedure should be improved before applying it to humans.

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