Neuronal targeted AAV Micro-Dystrophin restores neurobehavioural co-morbidities, grip strength and motor coordination in mdx52 mouse model of Duchenne Muscular Dystrophy
Konstantina Tetorou, Mónica Rebeca Gil-Garzon, Darren Chambers, Mustafa Görkem Özyurt, Filipe Nascimento, Wing Sum Chu, Simon N. Waddington, Benjamin W. Jarvis, Alex Kavanagh, Nicha Songsilph, Joanne Ng, Francesco Muntoni A
This study evaluates neuronal-targeted AAV micro-dystrophin therapy in mdx52 mice, showing IV delivery improves neurobehavioural, motor, and neuromuscular deficits more effectively than ICV delivery.
bioRxiv · May 26, 2026 · PreprintNeuroscience