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 proposed Wellstone Muscular Dystrophy Cooperative Research Center at the University of Texas Southwestern Medical Center (UTSW Wellstone MDCRC) is uniquely poised to begin translation of this technology to benefit patients with Duchenne muscular dystrophy, and ultimately, other forms of muscular dystrophy. For this to become a reality, it is essential to train a new generation of clinicians, physician-scientists, and basic scientists who will be able to apply these evolving technologies in the service of muscular dystrophy patients and their community. The objective of the Training Core is to 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. Our approach will be three fold: (1) To provide mentoring and financial support for two competitive, pilot and feasibility projects each year focused on advancing our understanding or treatment of any of the diverse forms of muscular dystrophy; (2) To develop and coordinate educational opportunities that will benefit all investigators and trainees associated with the Wellstone Center by both complementing and leveraging the strengths of existing training programs here at UTSW; (3) To foster avenues of interaction that will facilitate communication, collaboration, and synergy between the investigators at the UTSW Wellstone Center and those at other Wellston MDCRC network sites.
| 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 |
| Liu, Ning; Garry, Glynnis A; Li, Stephen et al. (2017) A Twist2-dependent progenitor cell contributes to adult skeletal muscle. Nat Cell Biol 19:202-213 |
| 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 |
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