Scholars Research Training Program in Regenerative Medicine, Gene Therapy, and Stem Cell Research

The UCSF CIRM Scholars Research Program is designed to equip the next generation of basic and clinical scientists with the expertise and motivation to make major strides in the fields of regenerative medicine, stem cell research, and gene therapy. To achieve this goal and fill the gap of needed experts in these fields in California requires robust training at multiple levels of education. This past year, the program selected four graduate students, seven post-doctoral fellows, and two clinical fellows from a large and highly competitive pool of applicants. Applicants were selected based on their prior work, proposed research, and potential for impact on the stem cell, gene therapy, and regenerative medicine fields. The selected applicants represent a diverse pool of backgrounds and specialties from stem cells to tissue engineering to gene editing all working toward a shared goal of improving human health. They each have established well thought out research and training programs under the guidance of strong and committed mentors. In addition to their individualized research plans, the trainees have been engaged in a series of community and patient oriented activities. For example, trainees have given presentations to local high school students, participated in the Bay Area Science Fair, and put on first UCSF "CIRM Days" aimed at educating and engaging high school students and undergraduates in the possibilities of stem cells and gene editing in improving human health. The trainees have also had the opportunity to directly engage with patients and their doctors who have been treated with or used stem cell/ gene editing based therapies to tackle specific diseases such as sickle cell , alpha thalassemia, severe combined immunodeficiency, and cancer. Research wise, the trainees have made much progress in their projects, which is shared with the other trainees and their mentors in a monthly research-in-progress meeting. The trainees are making inroads into areas such as engineering liver tissue, converting unhealthy fat to healthy fat, developing new cell therapies to target leukemia, engineering islets for treatment of diabetes, developing tumor cellular vaccines, improving genetic editing approaches, and much more. The trainees have also taken individualized courses specific to their research and attended conferences to both present their work as well as keep abreast of the latest conceptual, technical, and translational advances in the field. For example, many of the trainees attended and presented at the annual International Society of Stem Cell Research meeting this past summer. While we are only in the first year of the program, we are highly enthusiastic of the progress made by our trainees. They are well on the path to be future leaders in the stem cell/ gene therapy fields with great potential to positively impact patient care.

The UCSF CIRM Scholars Research Program aims to support the next generation of basic and clinical scientists in their journey toward advancing in the fields of regenerative medicine, stem cell research, and gene therapy. To achieve this goal and generate these experts for the advancement of science in California, we must support trainees at multiple levels of education. This past year, the program selected one clinical fellow and two postdoctoral fellows from a highly competitive pool of applicants. Our trainees were ultimately selected based on their prior work, proposed research, and potential for impact on the fields of stem cell, gene therapy, and regenerative medicine. The cohort for this year combines these new candidates with our trainees continuing from the first year of the grant. Overall, our cohort this year represents a diverse pool of backgrounds and specialties from stem cells to tissue engineering to gene editing all working toward a shared goal of improving human health. They each have established, rigorous research and training programs under the guidance of committed mentors. In addition to their individualized research plans, the trainees have been engaged in a series of community and patient oriented activities. For example, our trainees have independently mentored and tutored to students from backgrounds underrepresented in the sciences at multiple education levels (from high school to masters students). A majority of our trainees participated in UCSF’s “CIRM Days,” offering tours and talks aimed illuminating the possibilities of stem cell and gene editing therapies to high school students and undergraduate students. The trainees have also had the opportunity to directly engage with physicians and patients about their experience with stem cell/ gene editing based therapies for diseases such as sickle cell , alpha thalassemia, severe combined immunodeficiency, and cancer. In their second year, the trainees have persistently made impressive strides in their research projects that model human diseases and devise innovative therapeutic strategies and tools to combat these conditions. Collectively, they are addressing a wide spectrum of diseases including leukemia, retinal disorders, diabetes, neural degeneration, liver insufficiencies, and obesity. Research progress was shared among the entire cohort and their mentors in a monthly Research-In-Progress (RIPS) meeting. The trainees have also taken individualized courses specific to their research and attended conferences to both present their work as well as keep abreast of the latest conceptual, technical, and translational advances in the field. Three of the trainees who have recently left our training program will start or have already started faculty positions at other institutions, including UT Austin and UC San Diego. Two of our trainees have developed inventions from their research and one has published a paper in Cell Stem Cell. We excited to see the progress of this year and future cohorts as the grant progresses.

The UCSF CIRM Scholars Research Program aims to support the next generation of basic and clinical scientists in their journey toward advancing in the fields of regenerative medicine, stem cell research, and gene therapy. To achieve this goal and generate these experts for the advancement of science in California, we support trainees at multiple levels of education. During the past year, we supported 14 trainees including 4 graduate students, 8 postdoctoral fellows, and 2 clinical fellows. Twelve of these trainees are in their first year of this program and had been selected in the previous reporting period in a highly competitive application process which focused on their prior achievements proposed research, and potential for impact on the fields of stem cell, gene therapy, and regenerative medicine. The other two trainees including one post-doctoral and one clinical fellow were in their second year having been selected through a similar process in 2022. One of our trainees was recently awarded a grant, freeing up funds to allow us to appoint another postdoc, the most highly ranked alternate from our application cycle in Fall of 2023. The cohort represents a diverse pool of backgrounds and specialties from stem cells to tissue engineering to gene editing all working toward a shared goal of improving human health. They each have established, rigorous research and training programs under the guidance of committed mentors. In addition to their individualized research plans, the trainees have been engaged in a series of community and patient oriented activities. For example, our trainees have independently mentored and tutored students from backgrounds underrepresented in the sciences at multiple education levels (from high school to master’s students). Many of our trainees participated in our program’s “Stem Cell Tours,” offering tours and talks aimed illuminating the possibilities of stem cell and gene editing therapies to high school students and undergraduate students, many of whom come from backgrounds and groups underrepresented in science. The trainees have also had the opportunity to directly engage with physicians and patients about their experience with stem cell/ gene editing therapies for diseases such as type 1 diabetes, immune system disorders, and Duchenne muscular dystrophy. During this past year, the trainees have made impressive strides in their research projects that model human diseases and devise innovative therapeutic strategies and tools to combat these conditions. Collectively, they are addressing a wide spectrum of diseases including Alzheimer’s disease, ischemic injuries, type 1 diabetes, and cancer, among others. Research progress was shared among the entire cohort and their mentors in a monthly Research-In-Progress (RIPS) meeting. The trainees have also taken individualized courses specific to their research and attended conferences to both present their work as well as keep abreast of the latest conceptual, technical, and translational advances in the field. Two of our recent trainees have developed inventions from their research and one has published a paper in Cell Stem Cell. This year, six trainees presented posters or abstracts at scientific conferences, and one published a paper in Science Advances. We excited to see the progress of this year and future cohorts as the grant progresses.