Biology and Biotechnology of Cell and Gene Therapy

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Project Summary Graduate education has traditionally been successful in educating students in either engineering or the biomedical sciences, but the disparate nature of the scientific and engineering backgrounds necessary to successfully move the gene and cell therapy field forward requires novel educational approaches and methods that integrate these disciplines. With small molecule and protein therapies well-established in the pharmaceutical and biotechnology industries, gene and cell therapy represent the next generation of therapeutics to address serious unmet medical need. Because these therapeutics involve the delivery of DNA – in the form of genes or entire genomes – they have the potential to provide long-term therapeutic benefit following a single administration. However, the gene and cell therapy field face complex biological and technological challenges. Delivery of genetic constructs, either in vitro or in vivo, must be improved, and in addition therapeutic payloads including CRISPR/Cas9 and other genome editing machinery requires improved potency. In addition, cellular targets including human embryonic stem cells (hESCs), induced pluripotent stem cells (iPSCs), and adult stem cells are difficult to precisely control, since the repertoire of signals and cues that naturally control stem cell self-renewal and differentiation are not well understood, yet precise control is essential to unlocking applications in tissue engineering and regenerative medicine. The University of California at Berkeley has developed the highly interdisciplinary Biology and Biotechnology of Cell and Gene Therapy (BBCGT) Training Program. With the involvement and support of our 24 faculty, the Berkeley Stem Cell Center, Bioengineering Department, Molecular and Cell Biology Department, and Helen Wills Neuroscience Institute, we have designed and have been successfully implementing a program to support the education and training of predoctoral fellows in gene and cell therapy. This newly emerging discipline represents the convergence of the biological and biomedical sciences, physical sciences, engineering, and ethics. The primary objectives of our program have therefore been to formally organize the structure and scope of new training opportunities in this rapidly expanding discipline, to dissolve traditional academic barriers to interdisciplinary graduate science education, and to provide strong research training in academia and industry. As part of these efforts, we will immerse trainees in a Biology and Biotechnology of Cell and Gene Therapy curriculum, training in the responsible conduct of research, seminar series, annual retreat, interdisciplinary research, career development resources, industrial internship experience, and participation in a recruitment and retention plan to enhance diversity. The resulting program will be highly effective in training young scientists to work at the interface of the biomedical sciences and engineering in a rapidly-evolving, impactful, and timely area of biomedical research.