Systemic delivery of rAAV6-microdystrophin preserves muscle function and extends lifespan in a murine model of severe muscular dystrophy

Mice carrying mutations in both the dystrophin and utrophin genes die prematurely as a consequence of severe muscular dystrophy. Here, we demonstrate that intravascular administration of recombinant adeno-associated viral (rAAV) vectors carrying a microdystrophin gene restores dystrophin expression...

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Bibliografiset tiedot
Julkaisussa:Nat Med
Päätekijät: Gregorevic, Paul, Allen, James M., Minami, Elina, Blankinship, Michael J., Haraguchi, Miki, Meuse, Leonard, Finn, Eric, Adams, Marvin E., Froehner, Stanley C., Murry, Charles E., Chamberlain, Jeffrey S.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: 2006
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Article
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC4244883/
https://ncbi.nlm.nih.gov/pubmed/16819550
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/nm1439
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