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. Over the past four years the Wellstone Muscular Dystrophy Cooperative Research Center at the University of Texas Southwestern Medical Center (now the UTSW Wellstone MDSRC) has made tremendous strides toward the translation of this technology to benefit patients with Duchenne muscular dystrophy, and ultimately, other forms of muscular dystrophy. The mission of the Training Core has been just as impactful as it is essential to train a new generation of clinicians, physician-scientists, and basic scientists who are 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 of the Wellstone network in order to recruit, train, and maintain the next generation of transformative investigators focused on addressing the challenges of muscular dystrophy. Our approach focuses on five elements:
(Aim 1) To enhance the training environment of the UTSW Wellstone Center and promote trainee career advancement, (Aim 2) To recruit new trainees to muscular dystrophy research, (Aim 3) To mentor trainees in the process of obtaining funding support for the next step in their careers, (Aim 4) To organize research meetings involving Wellstone trainees, and (Aim 5) To organize training activities that benefit the overall muscular dystrophy research community. To accomplish these goals, we?ve built on the very successful program developed over the past four years of funding, that has both complemented and leveraged the strengths of existing training programs here at UTSW.
The goal of the UTSW Wellstone Training Core is to provide an environment in which trainees not only learn the technical aspects of genomic editing and their potential application to the treatment and study of Duchenne muscular dystrophy, but also become comfortable communicating and collaborating across disciplines from bedside to lab-bench and back again.