Research Training Program in Stem Cell Biology and Regenerative Medicine
Grant Award Details
Grant Type:
Grant Number:
EDUC4-12772
Investigator(s):
Award Value:
$5,234,731
Status:
Active
Progress Reports
Reporting Period:
Year 1
Reporting Period:
Year 2
Reporting Period:
Year 3
Grant Application Details
Application Title:
Research Training Program in Stem Cell Biology and Regenerative Medicine
Public Abstract:
The goal of our Research Training Program in Stem Cell Biology and Regenerative Medicine is to train the next generation of leaders in stem cell, gene therapy, and regenerative medicine, who understand the need to accelerate delivery of new therapies to patients with urgent, unmet needs; how to move new research discoveries from the bench to the bedside and of the complexities of implementing novel stem cell trials; and the ethical concerns surrounding stem cell research. We propose a training program for two 3-year cohorts, each with three predoctoral and six postdoctoral CIRM Scholars. This program will leverage the strengths of our institution in stem cell biology, regenerative medicine, and in conduct of innovative clinical trials. Scholars will undertake a program of: (1) formal course work in fundamental biology of stem cells and regenerative medicine; translation of stem cell therapy into clinical practice; and ethical issues emerging from this research and its implementation; (2) mentored independent research; (3) participation in patient outreach and education activities; and (4) community outreach through integration in ongoing programs. The training program will also feature a Stem Cell Research Day focused on Scholar research and an annual Stem Cell Biology, Gene Therapy, and Regenerative Medicine Symposium. Thirty-two research faculty will participate as mentors across several programs bridging basic science and clinical practice in different areas of stem cell research, including: hematopoietic cell transplantation, transplantation of insulin-secreting beta cells, gene therapy for repair of dysfunctional cell populations, directed differentiation of stem cells for repair/regeneration of damaged tissues, and cell-based immunotherapies. Through these activities and in this rich scientific and clinical environment, the proposed program will stimulate innovative multidisciplinary research that has potential to treat diseases that are an economic burden to the health care system such as diabetes, blood disorders, including sickle cell disease and blood cancers, Alzheimer’s disease and other neurological conditions, and many other disorders. Importantly, through the planned patient and community engagement and outreach activities we will promote in our Scholars an awareness of the inequities that impact implementation of therapies for all patients and will encourage Scholars to be active participants in better meeting the needs of patients in underrepresented and underserved communities. In addition, we will recruit Scholars who represent the diversity within the population of California. In conclusion, our training program will foster the early career development of outstanding scientists who are prepared to advance CIRM’s mission of accelerating novel stem cell-based therapies to treat chronic and incurable diseases.
Statement of Benefit to California:
The fields of stem cell research, gene therapy, and regenerative medicine are rapidly expanding and promise to have a significant impact on the healthcare and the economy of states and countries. We share with CIRM the mission of accelerating stem cell therapies to meet the medical needs of the people of California.
Our Institution is committed to being proactive in fostering an inclusive and safe environment that embraces diversity and diverse perspectives. Biomedical research flourishes when students, faculty, and staff are empowered to think big and to work inclusively. We are certain that diversity drives discovery and innovation. Our program is inspired by these ideals and leverages the strengths of our institution in stem cell biology and regenerative medicine, and its unique ability to develop and translate innovation into clinical trials, particularly into first-in-human studies. We are committed to training the next generation of leaders in the stem cell, gene therapy, and regenerative medicine fields, while enhancing their understanding of patients’ needs and of the complexities of implementing novel stem cell trials.
Our training program will benefit the citizens of California in many ways:
-By stimulating innovative multidisciplinary research that has the potential to treat diseases that are a psychological and economic burden to the health care system of California, such as diabetes, sickle cell disease and other blood disorders, Alzheimer’s disease and other neurological conditions, and many other disorders.
-By fostering a generation of innovators who are aware of the inequities that impact implementation of therapies for all and who will be active participants in meeting the needs of patients in underserved communities. Of particular importance, focus will be placed on developing and adopting technologies that can significantly reduce costs, so that novel cell and gene therapies can be available to all patients who need them, while reducing the economic burden on the California’s healthcare system.
-By recruiting and training a diverse workforce representing Californians who are underrepresented in medicine, including individuals who are first to attend college or from socioeconomically disadvantaged or underrepresented communities.
- By strengthening a workforce committed to accelerating the development and clinical testing of new stem cell and gene therapies and with the mission of educating the citizens of California about the benefits of stem cell research.
In conclusion, our training program will train future leaders in how to develop innovative approaches for the treatment of incurable diseases, as well as the need for interventions that address health inequities in underrepresented and undeserved communities, ultimately having tremendous impact on the lives of individual patients and their diverse communities in California.
Publications
- iScience (2024): Activation of ductal progenitor-like cells from adult human pancreas requires extracellular matrix protein signaling. (PubMed: 38433896)
- Blood (2024): A CD38-directed, single-chain T-cell engager targets leukemia stem cells through IFN-gamma-induced CD38 expression. (PubMed: 38394668)
- Int J Mol Sci (2024): Circadian Clock in Muscle Disease Etiology and Therapeutic Potential for Duchenne Muscular Dystrophy. (PubMed: 38731986)
- Cell Stem Cell (2023): Context matters: hPSC-derived microglia thrive in a humanized brain environment in vivo. (PubMed: 37419102)
- Elife (2023): Coordination between ECM and cell-cell adhesion regulates the development of islet aggregation, architecture, and functional maturation. (PubMed: 37610090)
- Diabetologia (2024): Differential CpG methylation at Nnat in the early establishment of beta cell heterogeneity. (PubMed: 38512414)
- Methods Mol Biol (2024): Directed Differentiation of Neurons from Human iPSCs for Modeling Neurological Disorders. (PubMed: 38630226)
- Cell Rep Methods (2023): Engineering CpG island DNA methylation in pluripotent cells through synthetic CpG-free ssDNA insertion. (PubMed: 37323577)
- Cancers (Basel) (2023): Evaluation of the Elements of Short Hairpin RNAs in Developing shRNA-Containing CAR T Cells. (PubMed: 37345185)
- Noncoding RNA (2023): Genetic Deletion of the LINC00520 Homolog in Mouse Aggravates Angiotensin II-Induced Hypertension. (PubMed: 37218991)
- Cell Stem Cell (2024): Human anti-PSCA CAR macrophages possess potent antitumor activity against pancreatic cancer. (PubMed: 38663406)
- Signal Transduct Target Ther (2024): Induced pluripotent stem cells (iPSCs): molecular mechanisms of induction and applications. (PubMed: 38670977)
- Nat Commun (2022): Integration of single-cell transcriptomes and biological function reveals distinct behavioral patterns in bone marrow endothelium. (PubMed: 36433940)
- J Vis Exp (2024): Isolation of Mouse Pancreatic Endothelial Cells. (PubMed: 38975772)
- J Clin Invest (2023): Long noncoding RNA LEENE promotes angiogenesis and ischemic recovery in diabetes models. (PubMed: 36512424)
- Elife (2024): Luminal epithelial cells integrate variable responses to aging into stereotypical changes that underlie breast cancer susceptibility. (PubMed: 39545637)
- Front Cell Neurosci (2023): Modeling brain macrophage biology and neurodegenerative diseases using human iPSC-derived neuroimmune organoids. (PubMed: 37342768)
- Front Neurosci (2023): Myelin organoids for the study of Alzheimer’s disease. (PubMed: 37942133)
- Trends Mol Med (2023): Pushing the boundaries of brain organoids to study Alzheimer’s disease. (PubMed: 37353408)
- Elife (2024): Regulation of nuclear transcription by mitochondrial RNA in endothelial cells. (PubMed: 38251974)
- Trends Pharmacol Sci (2024): The rise of epitranscriptomics: recent developments and future directions. (PubMed: 38103979)
- Cancer Treat Res (2023): RNA Modifications in Cancer Stem Cell Biology. (PubMed: 38112998)
- Cardiovasc Diabetol (2024): Role of long noncoding RNAs in diabetes-associated peripheral arterial disease. (PubMed: 39049097)
- Biochem Soc Trans (2024): Safeguarding genomic integrity in beta-cells: implications for beta-cell differentiation, growth, and dysfunction. (PubMed: 39364746)
- Breast Cancer Res (2023): Sustained postconfluent culture of human mammary epithelial cells enriches for luminal and c-Kit+ subtypes. (PubMed: 36653787)