From the smallest virus to the biggest gene: marching towards gene therapy for duchenne muscular dystrophy

Discov Med. 2006 Jun;6(33):103-8.

Abstract

The gene encoding dystrophin, which is altered in Duchenne muscular dystrophy, is one of the largest in mammalian species. Adeno-associated virus (AAV) is the smallest viral gene delivery vector with a "cargo" capacity of 6 kb and is the most effective gene delivery vehicle to muscle cells. Overlapping and trans-splicing AAV vector approaches are devised to do the "impossible."

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Dependovirus / genetics
  • Genetic Therapy / methods*
  • Genetic Vectors / genetics
  • Humans
  • Models, Biological
  • Muscular Dystrophy, Duchenne / genetics
  • Muscular Dystrophy, Duchenne / therapy*