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.
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| Stoller, Douglas; Araj, Faris; Amin, Alpesh et al. (2017) Implantation of a left ventricular assist device to provide long-term support for end-stage Duchenne muscular dystrophy-associated cardiomyopathy. ESC Heart Fail 4:379-383 |
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