The specific aim of this proposal is to provide advanced training to clinician scientists to pursue an academic career in translational research related to gene and stem cell based therapies for muscular dystrophy. The primary goal is to train physicians to conduct clinical gene therapy trials targeting muscular dystrophies. Most physicians at the completion of their medical or surgical training simply do not possess the knowledge or skills needed to implement a basic clinical trial, let alone one utilizing gene therapy vectors. Thus, a training program to accomplish these goals must provide exposure to the conduct of a clinical trial, data analysis, medical ethics, and regulatory affairs pertaining to human gene therapy, as well as basic molecular biology skills. We have designed a two-year stem and gene therapy training program for two Clinician Scientist trainees per year. We have assembled a diverse group of faculty members that include clinical investigators, molecular biologists, gene therapists, immunologists, pharmacologists, bioengineers, epidemiologists, statisticians, and bioethicists. During the first year of the training program, the Clinician Scientist trainees will participate in the NIH-funded Clinical Research Training Program (CRTP) established at the University of Pittsburgh. The CRTP aims to teach the skills necessary to design and conduct high quality clinical research. We will utilize the core curriculum and integrated seminar series of the CRTP as part of the training plan to avoid unnecessary duplication of resources and to allow for immediate implementation. Also during the first year, each trainee will be assigned to one of the ongoing MDCRC projects to get early exposure to a gene or stem cell approaches for treating DMD. During the second year of the program, the trainees will participate in the management of active clinical trials through the PCRN and the GCRC. The Pittsburgh Clinical Research Network (PCRN) is a clinical research organization that provides site management services for the conduct of industry-sponsored research. The General Clinical Research Center (GCRC) provides the infrastructure and support staff required for the conduct of clinical research and gene therapy trials at the University of Pittsburgh. The second year of training will also include three Gene Therapy Research Modules with basic coursework and laboratory experience focusing on gene therapy issues.
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