Credit Text: 

M. Lawlor, H. Meng/ Medical College of Wisconsin


Shown is a cross-section of a quadriceps muscle from a dog with X-linked myotubular myopathy one month after infusion of an AAV8 vector carrying the MTM1 therapeutic gene. Single dose gene therapy reversed muscle pathology, restored muscle strength and improved survival in mouse and dog models of X-linked myotubular myopathy, a fatal pediatric disease that affects the entire musculature (Childers et al.).