Increased mtDNA Abundance and Improved Function in Human Barth Syndrome Patient Fibroblasts Following AAV-TAZ Gene Delivery

Barth syndrome (BTHS) is a rare, X-linked, mitochondrial disorder caused by mutations in the gene encoding tafazzin. BTHS results in cardiomyopathy, muscle fatigue, and neutropenia in patients. Tafazzin is responsible for remodeling cardiolipin, a key structural lipid of the inner mitochondrial memb...

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Udgivet i:Int J Mol Sci
Main Authors: Suzuki-Hatano, Silveli, Sriramvenugopal, Mughil, Ramanathan, Manash, Soustek, Meghan, Byrne, Barry J., Cade, W. Todd, Kang, Peter B., Pacak, Christina A.
Format: Artigo
Sprog:Inglês
Udgivet: MDPI 2019
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6678701/
https://ncbi.nlm.nih.gov/pubmed/31336787
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms20143416
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