Project 2

Translating “myoediting” to young men with DMD is the fundamental goal of the UT Southwestern Wellstone MDCRC. Thus, the Center’s central hypothesis is that genomic editing can genetically correct mutations within the human dystrophin gene. Therefore, in Project 2 we will focus on identifying patients with potential dystrophin mutations that can be corrected in vitro, while comprehensively assessing the molecular/clinical phenotypes of these patients.