Stem cell models of TAFAZZIN deficiency reveal novel tissue-specific pathologies in Barth Syndrome
Olivia Sniezek Carney, Kodi William Harris, Yvonne Wohlfarter, Kyuna Lee, Grant J. Butschek, Arianna Franca Anzmann, Steven Michael Claypool, Anne Hamacher‐Brady, Markus Andreas Keller, Hilary J. Vernon
This study uses stem cell models to investigate tissue-specific pathologies in Barth Syndrome caused by TAFAZZIN deficiency, identifying mitophagy defects and potential therapeutic targets.
Human Molecular Genetics · Oct 29, 2024Peer-reviewedCell Biology