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."
From the smallest virus to the biggest gene: marching towards gene therapy for duchenne muscular dystrophy. Publishing Authors By Initials
From the smallest virus to the biggest gene: marching towards gene therapy for duchenne muscular dystrophy. Journal Published:
PUBLICATION TYPE: Journal Article
Journal: Discovery medicine
VOLUME: 6
Page Numbers: 103-8
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ISSN: 1539-6509
DAY: 19
MONTH: Jun
YEAR: 2006
From the smallest virus to the biggest gene: marching towards gene therapy for duchenne muscular dystrophy. Information
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LANGUAGE: eng
NlmUniqueID: 101250006
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Grant and Affiliation Information for From the smallest virus to the biggest gene: marching towards gene therapy for duchenne muscular dystrophy.
AFFILIATION: Department of Molecular Microbiology and Immunology, The University of Missouri School of Medicine, One Hospital Drive, Columbia, MO 65212, USA.
Country: United States
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MEDLINETA: Discov Med
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