Stem cell models of TAFAZZIN deficiency reveal novel tissue-specific pathologies in Barth Syndrome
O. S. Carney, K. W. Harris, Y. Wohlfarter, K. Lee, G. Butschek, A. Anzmann, S. M. Claypool, A. Hamacher-Brady, M. A. Keller, H. 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.
bioRxiv · Apr 28, 2024Cell Biology