AAV-mediated Liver-specific MPV17 Expression Restores mtDNA Levels and Prevents Diet-induced Liver Failure

Mutations in human MPV17 cause a hepatocerebral form of mitochondrial DNA depletion syndrome (MDS) hallmarked by early-onset liver failure, leading to premature death. Liver transplantation and frequent feeding using slow-release carbohydrates are the only available therapies, although surviving pat...

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Bibliographic Details
Main Authors: Bottani, Emanuela, Giordano, Carla, Civiletto, Gabriele, Di Meo, Ivano, Auricchio, Alberto, Ciusani, Emilio, Marchet, Silvia, Lamperti, Costanza, d'Amati, Giulia, Viscomi, Carlo, Zeviani, Massimo
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2014
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Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3880585/
https://ncbi.nlm.nih.gov/pubmed/24247928
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.230
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