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

Increased mtDNA Abundance and Improved Function in Human Barth Syndrome Patient Fibroblasts Following AAV-<i>TAZ</i> 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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Bibliographic Details
Main Authors: Silveli Suzuki-Hatano, Mughil Sriramvenugopal, Manash Ramanathan, Meghan Soustek, Barry J. Byrne, W. Todd Cade, Peter B. Kang, Christina A. Pacak
Format: Article
Language:English
Published: MDPI AG 2019-07-01
Series:International Journal of Molecular Sciences
Subjects:
Barth syndrome
mitochondrial disease
mtDNA copy numbers
gene therapy
AAV
TMEM65
fibroblasts
Online Access:https://www.mdpi.com/1422-0067/20/14/3416

Internet

https://www.mdpi.com/1422-0067/20/14/3416

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