Gene therapy in a murine model of Methylmalonic Acidemia (MMA) using rAAV9 mediated gene delivery

Methylmalonic acidemia (MMA), an inherited metabolic disorder caused by deficient activity of methylmalonyl-CoA mutase (MUT), carries a poor prognosis for long-term survival. While administration of a recombinant adeno-associated virus serotype 8 vector (rAAV8) can rescue Mut(-/-) mice from neonatal...

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Main Authors: Sénac, J S, Chandler, R J, Sysol, J R, Li, L, Venditti, C P
Format: Artigo
Sprog:Inglês
Udgivet: 2011
Fag:
Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3382069/
https://ncbi.nlm.nih.gov/pubmed/21776024
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2011.108
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