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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Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2012
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3370259/
https://ncbi.nlm.nih.gov/pubmed/22395529
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.39
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