Liver-Directed Recombinant Adeno-Associated Viral Gene Delivery Rescues a Lethal Mouse Model of Methylmalonic Acidemia and Provides Long-Term Phenotypic Correction

Methylmalonic acidemia is a severe metabolic disorder caused by a deficiency of the ubiquitously expressed mitochondrial enzyme, methylmalonyl-CoA mutase (MUT). Liver transplantation has been used to treat a small number of patients with variable success, and whether liver-directed gene therapy migh...

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Bibliografiske detaljer
Main Authors: Carrillo-Carrasco, Nuria, Chandler, Randy J., Chandrasekaran, Suma, Venditti, Charles P.
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc. 2010
Fag:
Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2936498/
https://ncbi.nlm.nih.gov/pubmed/20486773
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2010.008
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