Perm1 Gene Therapy Mitigates PRDM16-Associated Cardiomyopathy
Omid Mohammad Tavakoli‐Rouzbehani, Sophie L. Stephens, Benjamin Werbner, Marta W. Szulik, Sun Bo, Mira Hua, Shinya Watanabe, Ava Leonelli, Michael Goodman, Ryan D. Bia, Crystal F. Davey, Martin G. Golkowski, Sarah Franklin, Andrew Paul Landstrom, Sihem Boudina
This study identifies PERM1 as a downstream target of PRDM16 and shows that Perm1 gene therapy improves cardiomyopathy in Prdm16 knockout mice through mitochondrial and sarcomere preservation.
bioRxiv · Apr 15, 2026 · PreprintPhysiology