Cell BiologyHuman Molecular GeneticsPeer-reviewed
Stem cell models of TAFAZZIN deficiency reveal novel tissue-specific pathologies in Barth Syndrome
Olivia Sniezek Carney, Kodi William Harris, Yvonne Wohlfarter…·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.