Allele-selective knockdown of mutant DNAJB6 improves molecular features in a primary mouse myotube model of LGMDD1
A. Vohra, M. Haller, M. M. Paing, A. B. L. Dam, J. A. Daw, R. Bengoechea, S. K. Pittman, T.-Y. Wang, C. M. Yuede, T. M. Miller, H. L. True, S. Q. Harper, T.-F. Chou, C. C. Weihl, Andrew R Findlay
This study uses allele-selective knockdown to reduce mutant DNAJB6 in a mouse model of LGMDD1, improving molecular features associated with the disease.
bioRxiv · Nov 9, 2026 · PreprintNeuroscience