The Administrative Core of the UT Southwestern Wellstone Muscular Dystrophy Cooperative Research Center (MDCRC) will be responsible for the management and administration of the overall Center. The primary goal of the Administrative Core is to ensure our Center is successful in optimizing genome editing or myoediting as a feasible and practical approach to treating and even curing 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. The Administrative Core has set four fundamental goals to achieve during the terms of this NIH-funded U54 grant and they are as follows: 1. To provide effective leadership and management of the UT Southwestern Wellstone MDCRC. 2. To promote effective communication amongst the Center?s investigators as well as with investigators outside UT Southwestern, including members of the Wellstone Network Consortium. 3. To promote outreach to muscular dystrophy patients and their families. 4. To promote the enrollment of muscular dystrophy patients in clinical studies/trials. Therefore, 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 Cooperative 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. The successful completion of these Specific Aims will enhance the success of the UT Southwestern Wellstone MDCRC in achieving its goal of establishing genome editing as a new and innovative therapeutic modality in treating and possibly even curing DMD. Thus, the proposed NIH U54 Grant Application is relevant to and in keeping with the mission of the NIH.
|Makarewich, Catherine A; Munir, Amir Z; Schiattarella, Gabriele G et al. (2018) The DWORF micropeptide enhances contractility and prevents heart failure in a mouse model of dilated cardiomyopathy. Elife 7:|
|Long, Chengzu; Li, Hui; Tiburcy, Malte et al. (2018) Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing. Sci Adv 4:eaap9004|
|Amoasii, Leonela; Hildyard, John C W; Li, Hui et al. (2018) Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy. Science 362:86-91|
|Zhang, Yu; Long, Chengzu; Bassel-Duby, Rhonda et al. (2018) Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing. Physiol Rev 98:1205-1240|
|Amoasii, Leonela; Olson, Eric N; Bassel-Duby, Rhonda (2018) Control of Muscle Metabolism by the Mediator Complex. Cold Spring Harb Perspect Med 8:|
|Hashimoto, Hisayuki; Olson, Eric N; Bassel-Duby, Rhonda (2018) Therapeutic approaches for cardiac regeneration and repair. Nat Rev Cardiol 15:585-600|
|Makarewich, Catherine A; Baskin, Kedryn K; Munir, Amir Z et al. (2018) MOXI Is a Mitochondrial Micropeptide That Enhances Fatty Acid ?-Oxidation. Cell Rep 23:3701-3709|
|Bi, Pengpeng; McAnally, John R; Shelton, John M et al. (2018) Fusogenic micropeptide Myomixer is essential for satellite cell fusion and muscle regeneration. Proc Natl Acad Sci U S A 115:3864-3869|
|Zhang, Yu; Long, Chengzu; Li, Hui et al. (2017) CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice. Sci Adv 3:e1602814|
|Makarewich, Catherine A; Olson, Eric N (2017) Mining for Micropeptides. Trends Cell Biol 27:685-696|
Showing the most recent 10 out of 37 publications