CIRM Scholars Comprehensive Research Training Program
Grant Award Details
Grant Type:
Grant Number:
EDUC4-12822
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:
CIRM Scholars Comprehensive Research Training Program
Public Abstract:
We will deliver a comprehensive doctoral, postdoctoral and clinical researcher training program designed to develop the current and next generation of researchers in the fields of stem cell biology, gene therapy and regenerative medicine. Our program is centered around the comprehensive understanding, use and manipulation of stem and progenitor cells, cells which promise to revolutionize the way that human diseases and disorders are treated. Advancing the goals of CIRM to develop new treatments for human disease based on stem and progenitor cells will require the understanding and application of multiple technologies. Researchers in this field will need to understand multiple disciplines and participate in multi-disciplinary research teams where each of the participants understands the capabilities and shortcomings of each other's technologies. Trainees will be recruited from within existing labs and by external recruitment with the goal of recruiting a diverse cohort of young scientists. Our stem cell training program will emphasize broad, cross-disciplinary training, exposing trainees to concepts and techniques in diverse fields such as stem cell biology, biomedical engineering, pre-clinical development and clinical practice. Didactic courses are tailored to address the needs of the researchers of the future. All incoming trainees will have the opportunity to learn the ethical conduct of hypothesis driven research, understand the concepts of reproducibility and rigor, know advanced techniques in cell, DNA, RNA and protein analysis and participate in team building exercises in hypothesis driven research. A new course on the ethical, legal and societal implications of stem cell research will be offered for trainees at all levels. Journal clubs will allow fellows to keep abreast of the latest developments in the field. In addition, we will host monthly seminars where experts present the latest developments in multiple fields related to stem cell research, gene therapy and regenerative medicine including developmental biology, bioengineering, molecular biology, etc. In this way fellows can be updated on the latest developments in the field broadly defined. Lectures will be recorded for web access so that topics can be revisited at leisure and accessed by the entire stem cell research community. Events carried out throughout the year will expose trainees first-hand to patients and disease advocates, in order to discuss the real-world challenges facing treatment. Trainees will be coached in communications skills so as to help in their outreach within the scientific, lay and patient communities. An annual retreat will allow the program administration to determine progress of all trainees as well as allowing trainees to share their results with experts in the field and to develop networking skills. The overall goal will be to train researchers capable of carrying out multidisciplinary research and developing new treatments for new human disease.
Statement of Benefit to California:
A primary goal of Proposition 14 is to continue to translate basic stem cell research to clinical applications. The disability and loss of personal freedom and earning power resulting from a disease or disorder are devastating and create a financial burden for California in addition to the suffering caused to patients and their families. Therapies using stem cells have the potential to change millions of lives. Using stem cells as models of disease will help us understand the underlying causes of disease and likely aid in the development of drugs to treat those diseases. For the potential of stem cells to be realized, California researchers need the personnel to develop them into viable treatments. Therefore, the raison d’etre for the proposed program is to provide training to the next generation of researchers in stem cell biology, gene therapy and regenerative medicine capable of advancing the development of new methods of treating human disease. The breadth and depth of the stem cell biology, gene therapy and regenerative medicine research programs, which have already made important advances and secured significant funding from CIRM, will act as the core around which all training will be organized. Anticipated benefits of our Training Program to the Citizens of California include: creation of a training program that will attract the best and brightest minds to the state; development of new cell-based treatments for a variety of diseases and disorders; generation of new techniques for using stem cells (and derived cells) to deliver drugs or other agents to tissues, thereby developing new treatment methods; development of methods of using gene therapy to treat human diseases; improved methods for understanding normal development and environmental risks to the early embryo; improved methods for detecting and understanding effects of toxicants in the environment and workplace; improved clinical trial methodology that will directly impact human testing of stem cell and gene therapies; development of new improved methods for developing and testing drugs for treating disease; transfer of new technologies and intellectual property to the public realm with resulting IP revenues coming into the state; creation of new biotechnology spin-off companies based on generated intellectual property; creating interdisciplinary research teams that will have a competitive edge for obtaining funding from out of state; development of researchers and clinicians that will establish clinical research programs in the state; and, creation of new jobs in the biotechnology sector. It is anticipated that the return to the State in terms of revenue, health benefits for its Citizens, job creation, and revenue, will be significant in the long term.
Publications
- Cell Rep (2022): Absence of microglia promotes diverse pathologies and early lethality in Alzheimer’s disease mice. (PubMed: 35705056)
- J Invest Dermatol (2024): Ancestral Diversity of Skin Reaches Single-Cell Resolution. (PubMed: 39306776)
- Sci Rep (2024): Dendrite injury triggers neuroprotection in Drosophila models of neurodegenerative disease. (PubMed: 39433621)
- Neuron (2025): Distinct molecular patterns in R6/2 HD mouse brain: Insights from spatiotemporal transcriptomics. (PubMed: 40482637)
- Adv Biol (Weinh) (2023): DNA Methylation Dynamics During Esophageal Epithelial Regeneration Following Repair with Acellular Silk Fibroin Grafts in Rat. (PubMed: 36658732)
- Cold Spring Harb Perspect Biol (2023): Engineering Immunomodulatory Biomaterials to Drive Skin Wounds toward Regenerative Healing. (PubMed: 36123029)
- EMBO Mol Med (2023): Graft conditioning with fluticasone propionate reduces graft-versus-host disease upon allogeneic hematopoietic cell transplantation in mice. (PubMed: 37538042)
- Stem Cells (2023): How and Why the Circadian Clock Regulates Proliferation of Adult Epithelial Stem Cells. (PubMed: 36740940)
- J Invest Dermatol (2024): Integrated Single-Cell Analysis Reveals Spatially and Temporally Dynamic Heterogeneity in Fibroblast States during Wound Healing. (PubMed: 39019149)
- J Invest Dermatol (2023): Mapping Human Skin: One Sequenced Cell at a Time. (PubMed: 37747389)
- Neuron (2024): Microglia are not necessary for maintenance of blood-brain barrier properties in health, but PLX5622 alters brain endothelial cholesterol metabolism. (PubMed: 39142282)
- Nat Cell Biol (2023): Microglia promote anti-tumour immunity and suppress breast cancer brain metastasis. (PubMed: 37957324)
- Neuron (2024): Microglia protect against age-associated brain pathologies. (PubMed: 38897208)
- Antibodies (Basel) (2024): Ocular Mucous Membrane Pemphigoid Demonstrates a Distinct Autoantibody Profile from Those of Other Autoimmune Blistering Diseases: A Preliminary Study. (PubMed: 39584991)
- J Gen Physiol (2022): Piezo1 regulates cholesterol biosynthesis to influence neural stem cell fate during brain development. (PubMed: 36069933)
- Nat Genet (2023): Preneoplastic stromal cells promote BRCA1-mediated breast tumorigenesis. (PubMed: 36914836)
- J Am Acad Dermatol (2024): Response to Arbache et al., “Comment on ‘Vulvar lichen sclerosus and vitiligo: Overlap and clinical features'”. (PubMed: 38692434)
- J Invest Dermatol (2023): A Roadmap for a Consensus Human Skin Cell Atlas and Single-Cell Data Standardization. (PubMed: 37612031)
- Nature (2023): Signalling by senescent melanocytes hyperactivates hair growth. (PubMed: 37344645)
- iScience (2023): Single-nuclei transcriptome analysis of Huntington disease iPSC and mouse astrocytes implicates maturation and functional deficits. (PubMed: 36590162)
- Development (2023): SIX1+PAX3+ identify a progenitor for myogenic lineage commitment from hPSCs. (PubMed: 37366057)
- Nature (2023): A spatially resolved single-cell genomic atlas of the adult human breast. (PubMed: 37380767)
- APL Bioeng (2023): Targeting the ADPKD methylome using nanoparticle-mediated combination therapy. (PubMed: 37305656)
- Nat Commun (2024): tauFisher predicts circadian time from a single sample of bulk and single-cell pseudobulk transcriptomic data. (PubMed: 38714698)
- Neuron (2024): Therapeutic potential of human microglia transplantation in a chimeric model of CSF1R-related leukoencephalopathy. (PubMed: 38897209)
- bioRxiv (2024): Uncovering Minimal Pathways in Melanoma Initiation. (PubMed: 38106189)
- Biomicrofluidics (2023): Vascularized liver-on-a-chip model to investigate nicotine-induced dysfunction. (PubMed: 38155919)
- bioRxiv (2024): Visualizing PIEZO1 Localization and Activity in hiPSC-Derived Single Cells and Organoids with HaloTag Technology. (PubMed: 38187535)
- JAAD Int (2024): Vulvar dermatoses and depression: A systematic review of vulvar lichen sclerosus, lichen planus, and lichen simplex chronicus. (PubMed: 38371668)