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

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Grant Award Details

Grant Number:
EDUC4-12812
Investigator(s):
Award Value:
$5,373,742
Status:
Active

Progress Reports

Reporting Period:
Year 1
Reporting Period:
Year 2
Reporting Period:
Year 3

Grant Application Details

Application Title:

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

Public Abstract:
The proposed 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. The focus of our program is graduate students, post-doctoral fellows, and clinical fellows. Trainees from each of these pools will be selected from diverse ethnic backgrounds based on their prior scholarship, their mentor, and their proposed research. Scholars will be selected from a wide range of disciplines such as cell and molecular biology, gene editing, immunology, organismal biology, computational biology, synthetic biology, bioengineering, therapeutic sciences, and clinical trials. They will perform their research under the mentorship of our institution’s world class faculty in regenerative medicine, stem cell research and gene therapy. They will have access to the remarkable physical and intellectual infrastructure of the institution. In addition to funding the selected trainees’ research, the proposed program includes a cutting-edge curriculum combining coursework, community outreach, education activities, and career mentoring. Coursework about the science, clinical application, and ethics of stem cells, gene therapy, and regenerative medicine will be complemented by rich opportunities for in-depth learning about specific diseases through conversations with patients, seminars and informal discussions of stem cell and gene therapy research and clinical strategies with scientists and physicians, as well as debates about the ethical, legal, and social implications with leading bioethicists. Scholars will also gain important skills as educators by teaching high school students, undergraduates, colleagues, and the broader community about the potential of stem cell and gene therapy in improving the population’s health. Scholars will be closely mentored with multiple opportunities to gain training in skills that will enable them to pursue a broad array of career paths from academic research to industry-based product development to clinical practice to policy, all critical to enabling the promise of regenerative medicine. Emphasis will be placed on promoting diversity, equity, and inclusion among the scholars through multiple dedicated efforts at recruitment and retention. The strength of our institution’s training environment, as evidenced by our track record with prior trainees, combined with the strength of the proposed program, should ensure success at producing leaders in all these areas.
Statement of Benefit to California:
We envision that the citizens of the state of California will benefit in many ways from our university’s proposed Scholars Training Program for graduate students, postdoctoral fellows, and clinical scientists. Collectively, the basic research, translational strategies, and clinical therapies that emerge from the work of our university’s California Institute for Regenerative Medicine (CIRM) funded trainees will be an important stimulus to the state economy, particularly the biotechnology sector and associated medical enterprises. Additionally, specific groups of individuals will directly benefit from work that is focused on cell-based therapies for repairing tissues and organs whose damage leads to common medical conditions, for example, diabetes, cardiovascular disease, Parkinson’s disease, paralysis and/or immune dysfunction. On the way to achieving the CIRM’s ultimate goals in terms of novel regenerative therapies for patients, we envision that numerous other benefits will emerge. For example, human embryonic stem cell (hESC) systems are powerful tools for unraveling the molecular basis of human development, which remains largely a black box. A fundamental lack of understanding regarding the mechanisms that give rise to the hundreds of cell types that form tissues and organs makes it extremely difficult to discern why these processes sometimes go awry, leading to birth defects and/or setting the stage for many diseases. Additionally, novel therapies for other medical conditions are also likely to emerge. In this regard, some forms of cancer are now thought to be associated with the proliferation of stem cells that carry mutations in genes that promote their self-renewal, rather than differentiation and integration into the compartment that they normally occupy. Other important applications include drug development. For example, hESCs and their differentiated progeny could be used to screen promising compounds for efficacy, safety and/or toxicity. Where will the workforce come from that will enable this revolution in how the medical establishment approaches patient care? Stem cell and gene therapy is a rapidly growing field that must be rapidly populated with scientists and clinicians who are specially trained in all aspects of regenerative medicine, a new specialty. This necessity makes the funding of CIRM-sponsored training programs especially critical for institutions such as ours that have the ability to make important research discoveries and translate them into clinical therapies. Our university has a long and distinguished history of training leaders in science and/or medicine who easily traverse the boundaries between academia and industry. Our past successes strongly suggest that our CIRM-funded training programs will be equally successful. Accordingly, we expect that our trainees will become leaders in the field.

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