Functional protection of dystrophic mouse (mdx) muscles after adenovirus-mediated transfer of a dystrophin minigene

Proc Natl Acad Sci U S A. 1996 Apr 16;93(8):3570-4. doi: 10.1073/pnas.93.8.3570.

Abstract

Fast skeletal muscles of mdx (X chromosome-linked muscular dystrophy) mice were injected after birth with a recombinant adenovirus containing a minidys- trophin gene, a 6.3-kbp cDNA coding for the N- and C-terminal ends of dystrophin. Adult muscles were challenged by forced lengthening during tetanic contractions. Stretch-induced mechanical and histological damages were much reduced in injected muscles, in direct proportion of the Miniber of fibers expressing minidystrophin. Damaged fibers were preferentially found among minidystrophin-negative regions. Minidystrostrophin confers an important functional and structural protection of limb muscles against high mechanical stress, even after a partial somatic gene transfer.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenoviridae / genetics
  • Animals
  • Dystrophin / genetics*
  • Gene Transfer Techniques*
  • Genetic Therapy*
  • Genetic Vectors
  • In Vitro Techniques
  • Mice
  • Mice, Inbred mdx
  • Muscle Contraction
  • Muscle Fibers, Fast-Twitch / physiology
  • Muscle, Skeletal / pathology
  • Muscle, Skeletal / physiopathology
  • Muscular Dystrophy, Animal / genetics*
  • Muscular Dystrophy, Animal / physiopathology
  • Muscular Dystrophy, Animal / therapy*
  • Stress, Mechanical

Substances

  • Dystrophin