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Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy

Very long-chain acyl-coA dehydrogenase (VLCAD) is the rate-limiting step in mitochondrial fatty acid oxidation. VLCAD-deficient mice and patients clinical symptoms stem from not only an energy deficiency but also long-chain metabolite accumulations. VLCAD-deficient mice were treated systemically wit...

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Detalles Bibliográficos
Autores principales:	Keeler, Allison M, Conlon, Thomas, Walter, Glenn, Zeng, Huadong, Shaffer, Scott A, Dungtao, Fu, Erger, Kirsten, Cossette, Travis, Tang, Qiushi, Mueller, Christian, Flotte, Terence R
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2012
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3370259/ https://www.ncbi.nlm.nih.gov/pubmed/22395529 http://dx.doi.org/10.1038/mt.2012.39

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