The breakthrough technology of CRISPR/Cas9-mediated genomic editing offers an entirely new and powerful therapeutic potential for patients suffering from genetic diseases like Duchenne Muscular Dystrophy (DMD). Since joining the Wellstone Network in 2015, the UT Southwestern Myoediting Core has served both as an essential coordinating hub for the Wellstone research groups (Projects 1 & 2) at UT Southwestern and as a conduit for sharing expertise and resources with the broader scientific community. Moving forward, our goals for this Shared Research Resource Core are:
(Aim #1) To continue to support Projects 1 and 2 of the UT Southwestern Wellstone Center by providing the cutting-edge technologies of human iPSCs, CRISPR/Cas9 genomic editing, and cardiomyocyte differentiation.
(Aim #2) To help advance the study of muscular dystrophy worldwide by providing a DMD/BMD Biorepository of patient-derived DMD iPSCs and their corresponding CRISPR/Cas9-corrected isolates along with information regarding the clinical phenotype and genomic sequence of the donor patient.
(Aim #3) To help other investigators take full advantage of these resources by freely sharing our streamlined protocols and providing training (either on site, or in silico) in best practices and approaches. These resources are available to investigators from other Wellstone Centers as well as the general scientific community at large.
The Myoediting Core is designed and managed as a shared Scientific Research Resource Core to support the research of the Muscular Dystrophy Specialized Research Center (MDSRC) at UT Southwestern, other Wellstone MDSRCs, as well as serving as a resource for the national and international muscular dystrophy research community. This proposal offers a novel strategy for treating Duchenne muscular dystrophy, called ?Myoediting? which involves molecularly modifying the genome of muscle cells affected by this disease, correcting the mutation in a manner that causes permanent skipping of the mutant exons within the dystrophin gene transcript. The Myoediting Core is developing state-of- the-art myoediting tools, technologies and reagents to improve efficacy of current gene repair strategies and to facilitate new strategies.