The techniques of CRISPR/Cas9-mediated genomic editing and the ability to generate induced pluripotent stem cells (iPSCs) from a sample of a patient's blood have placed medicine on the brink of a revolution in our ability to treat, and perhaps even cure, a broad range of genome-based diseases. The goal of the UT Southwestern MDCRC is to improve treatment of Duchenne muscular dystrophy (DMD) by developing a new therapeutic strategy called myoediting based on these technologies. The Center will be built around five integral components. These include: two inter-related research projects (1) one that will work to optimize the tools for application of CRISPR/Cas9- mediated DMD exon skipping to permanently restore dystrophin function, and the other (2) that will identify genetic and biomarker associations with cardiac phenotypes in adult DMD patients and serve as a primary source for a Duchenne Skipper Database. These projects will be complemented and supported by three Cores (A) an Administrative Core, that will also direct patient outreach and education, (B) a Myoediting Core, which will develop DMD-in-a-dish cardiac and skeletal muscle disease models and house the Duchenne Skipper Database for designing guide RNAs, and (C) a Training Core, which will enhance the educational environment in order to recruit, train, and maintain the next generation of transformative investigators focused on addressing the challenges of muscular dystrophy. We firmly believe myoediting to be a unique once-in-a-lifetime opportunity to discover a functional cure for many thousands of patients and their families devastated by DMD.
The goal of the UT Southwestern MDCRC is to improve treatment of DMD by advancing a new and rapidly evolving technology that combines the CRISPR/Cas9 system of genomic editing with the ability to generate induced pluripotent stem cells (iPSCs). We have termed this process 'myoediting.'
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