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Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy

Methylmalonic acidemia (MMA) is an organic acidemia caused by deficient activity of the mitochondrial enzyme methylmalonyl-CoA mutase (MUT). This disorder is associated with lethal metabolic instability and carries a poor prognosis for long-term survival. A murine model of MMA that replicates a seve...

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Detalles Bibliográficos
Autores principales:	Chandler, Randy J, Venditti, Charles P
Formato:	Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2009
Materias:	Original Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2839224/ https://www.ncbi.nlm.nih.gov/pubmed/19861951 http://dx.doi.org/10.1038/mt.2009.247

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