for the Administrative Core The Administrative Core of the UT Southwestern Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center (MDSRC) will be responsible for the management and administration of the overall Center. The primary goal of the Administrative Core is to ensure our Center remains successful in optimizing genome editing or myoediting as a feasible and practical approach to treating Duchenne muscular dystrophy (DMD). Achievement of the Center?s goal will be a transformative moment in modern medicine and will be a significant contribution to the field of muscular dystrophy research as well as to patient care. In order for the UT Southwestern Wellstone MDSRC to remain successful in its scientific endeavors, the Administrative Core will continue to provide leadership amongst the various investigators within the Center in order to ensure efficient use of resources and the successful completion of the goals outlined in this NIH P50 Grant Application. The Administrative Core will provide an environment to facilitate the sharing and exchange of ideas and discoveries within the Center as well as with the scientific community at-large. The Center will also actively seek to disseminate these discoveries to patients and their families. The nurturing environment that exists at UT Southwestern will foster the training and education of the next generation of clinical and scientific investigators in the field of muscular dystrophy research. Finally, the UT Southwestern Administrative Core will continue its effective communication with the NIH and Wellstone Network Consortium in order to collectively advance the field of muscular dystrophy research forward. The goals set out by the Administrative Core will be accomplished by pursuing the following three specific aims:
Specific Aim 1 : To facilitate and enhance clinical and scientific interactions within the UT Southwestern Wellstone Muscular Dystrophy Specialized Research Center.
Specific Aim 2 : To promote the dissemination of knowledge, resources, and discoveries related to genome editing and Duchenne muscular dystrophy.
Specific Aim 3 : To serve as an advocate for and promote outreach to muscular dystrophy patients and their families regarding novel therapeutics and advances in clinical care. Under the continued effective leadership of the Administrative Core, the UT Southwestern Senator Paul D. Wellstone MDSRC will strive to achieve its overarching goal of optimizing genome editing to be an effective method of permanently restoring dystrophin expression within myocytes and thus provide a long awaited therapy for DMD. Thus, the proposed NIH P50 Grant Application is relevant to and in keeping with the mission of the NIH.
for the Administrative Core The proposed NIH P50 Grant Application has significant relevance to the public health of our nation. The overall study is both significant and innovative because it proposes that genome editing can permanently restore dystrophin expression within the skeletal muscle of patients with Duchenne muscular dystrophy. The Administrative Core will be charged with the mission of providing effective leadership and management of the UT Southwestern Muscular Dystrophy Specialized Research Center in order to enhance the Center?s opportunity to successfully and significantly contribute to the field of muscular dystrophy research and patient care.
