A Center for Stem Cell Disease Modeling and Therapeutics
Grant Award Details
Grant Type:
Grant Number:
INFR6.2-15527
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Cell Line Generation:
Award Value:
$3,999,997
Status:
Active
Grant Application Details
Application Title:
A Center for Stem Cell Disease Modeling and Therapeutics
Public Abstract:
The goal of this project is to support the discovery and evaluation of novel therapeutics using stem cell-based models and drug and CRISPR screening. The facility will provide California researchers access to tools, technologies, and resources for regenerative medicine research.
Statement of Benefit to California:
This project benefits California by helping accelerate research to find cures for major diseases of the nervous system, heart, and immune system. This project also provides training for Californians who want to enter the biotechnology workforce. Finally, the shared laboratory strengthens the California research community by providing advanced tools and technologies to a wide variety of regenerative medicine researchers.
Publications
- Nat Commun (2025): Characterizing and controlling CRISPR repair outcomes in nondividing human cells. (PubMed: 41249169)
- Cell Rep (2025): Dual alpha-globin-truncated erythropoietin receptor knockin restores hemoglobin production in alpha-thalassemia-derived erythroid cells. (PubMed: 39754719)
- bioRxiv (2026): EXCAVATE-HT: A Bioinformatic Pipeline to Identify Targetable Genomic Variants for Allele-Specific Editing. (PubMed: 42539204)
- medRxiv (2026): Leveraging human genetic variation to therapeutically target hundreds of genes with dominant & dispensable disease alleles. (PubMed: 41929339)