Adenoviral-mediated correction of methylmalonyl-CoA mutase deficiency in murine fibroblasts and human hepatocytes

BACKGROUND: Methylmalonic acidemia (MMA), a common organic aciduria, is caused by deficiency of the mitochondrial localized, 5'deoxyadenosylcobalamin dependent enzyme, methylmalonyl-CoA mutase (MUT). Liver transplantation in the absence of gross hepatic dysfunction provides supportive therapy a...

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Autores principales: Chandler, Randy J, Tsai, Matthew S, Dorko, Kenneth, Sloan, Jennifer, Korson, Mark, Freeman, Richard, Strom, Stephen, Venditti, Charles P
Formato: Artigo
Lenguaje:Inglês
Publicado: BioMed Central 2007
Materias:
Research Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC1876207/
https://ncbi.nlm.nih.gov/pubmed/17470278
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1471-2350-8-24
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