AAV-Delivered RNAi Targeting Mutant LDB3 Prevents and Reverses Myofibrillar Myopathy through Mechanosignaling Restoration
Pankaj Pathak, Jessica Palmeri, Jessica J. Hale, Anna Sabu-Kurian, Morteza Peiravi, Danielle Springer, Yan Li, KR Johnson, Ami K. Mankodi
This study shows that AAV-delivered RNAi targeting mutant LDB3 can reverse or prevent myofibrillar myopathy by restoring mechanosignaling pathways in mice.
bioRxiv · Mar 31, 2026 · PreprintNeuroscience