California's Stem Cell Agency

The Berkeley Stem Cell Center’s CIRM Scholars training program has the dual goals of training the next generation of leaders in regenerative medicine and simultaneously supporting cutting edge research in stem cell biology and gene therapy. Following a competitive fellowship application process the program appointed a diverse and talented initial cohort of CIRM Scholars, including two physicians and four PhDs. Several are focused on engineering improved gene therapy methods, including enabling the use of larger and more robust gene editing molecules, targeting these tools to specific cell types in the brain, and honing gene editing and transplantation strategies to cure sickle cell disease. Another fellow is developing methods to detect subtle but functionally significant differences in seemingly identical cells by simultaneously measuring levels of specific protein and messenger RNAs. Two fellows’ projects seek a deeper understanding of fundamental regulatory principles controlling embryogenesis and neural stem cell differentiation. The CIRM Scholars’ wide range of backgrounds and expertise contributes to a learning environment of scientific excellence and motivation to address currently unmet medical needs. In addition to intensive laboratory research the CIRM Scholars training program includes monthly fellows’ meetings and courses in stem cell biology, biomedical entrepreneurship and ethical, legal and social issues underlying development and deployment of stem cell and gene editing-based therapies. A formal seminar and discussion course covering modern approaches to stem cell biology, regenerative medicine and gene therapy was developed for this program and debuted during the past year. Also during the past year, CIRM Scholars presented their research at local and national meetings and enabled undergraduate students to experience scientific discovery firsthand by serving as laboratory mentors. The program seeks to improve in response to trainee and advisory committee feedback and looks forward to expanding to its full size by appointing its second cohort of trainees in early 2023.