Allele-selective knockdown of mutant DNAJB6 improves molecular features in a primary mouse myotube model of LGMDD1
Aliasgar Vohra, Meade Haller, May M Paing, Alina Bao Lan Dam, Jil A Daw, Rocio Bengoechea, Sara K Pittman, Ting-Yu Wang, Carla M Yuede, Timothy M Miller, Heather L True, Scott Q Harper, Tsui-Fen Chou, Conrad 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