This application from the Center for Cell and Gene Therapy (CAGT) / Baylor College of Medicine (BCM) describes a comprehensive training program in cellular and genetic therapies, for both graduate students and postdoctoral fellows. This burgeoning area of translational research remains deficient in comprehensive training schemes for individuals who wish to address basic and clinical translational aspects using these biological agents. A stimulating program of education and laboratory training from high quality mentors should continue to attract promising candidates, including under-represented minorities, to this emerging field and increase the likelihood that they will pursue careers as translational researchers. Translation of basic research discoveries to clinical practice has become increasingly complex, such that advances in basic research can only be efficiently implemented when teams of investigators collaborate to validate these novel strategies in the clinic. This type of translational research training requires the involvement of a dedicated team of mentors with diverse but complementary skills. The proposed training exploits the outstanding biomedical curricula and experienced faculty within BCM and CAGT. A cadre of 26 faculty members with research interests that include vector design and targeting, stem cell biology, molecular and cellular genetics, immunotherapy, gene therapy and stem cell transplantation will be available to mentor graduate students and both MD and PhD postdoctoral fellows. Major strengths of the program are its;(1) targeting of rigorous basic biomedical science and clinical research training toward a translational goal;(2) established and multilevel integration with the proven scientific excellence of cell biology and genetics research training programs at BCM;(3) involvement of mentors, including program director and co directors, who have a wealth of experience in translational research and in training young investigators (4) availability of unique resources such as the GMP laboratories for training. Augmenting the formal course work and laboratory training will be opportunities to present recent research findings at annual retreats sponsored by CAGT and academic departments within BCM. During the first 8 years of funding this approach has successfully trained 14 graduate students who have completed their PhD degrees: all remain in academic medicine. Eight postdoctoral fellows have completed training in translational cell and gene therapy research and all remain in the field with 5 appointed to academic faculty positions and 3 in biotechnology. Of all trainees who have completed training or who are still in training 9 are under- represented minorities and 3 have under-privileged backgrounds. The maintenance of support will allow us to continue to provide trainees with a strong foundation for translational research careers in this emerging area.

Public Health Relevance

This proposal aims to train young researchers so that they can move cell and gene based therapies from basic science laboratories and into the clinic. These therapies hold great promise for regenerative medicine, cancer and other disorders, but can only be implemented after the specialized training that this award will provide

National Institute of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
Institutional National Research Service Award (T32)
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NHLBI Institutional Training Mechanism Review Committee (NITM)
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Welniak, Lisbeth A
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Baylor College of Medicine
Schools of Medicine
United States
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