NeurosciencebioRxivPreprint
AAV-Delivered RNAi Targeting Mutant LDB3 Prevents and Reverses Myofibrillar Myopathy through Mechanosignaling Restoration
Pankaj Pathak, Jessica Palmeri, Jessica J. Hale…·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.
DOI 10.64898/2026.03.28.715031