From the smallest virus to the biggest gene: marching towards gene therapy for duchenne muscular dystrophy.
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."
Duan
Dongsheng
D
Department of Molecular Microbiology and Immunology, The University of Missouri School of Medicine, One Hospital Drive, Columbia, MO 65212, USA.
D000229:Dependovirus / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics; D006801:Humans; D008954:Models, Biological; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000628:therapy*