Base Editing, Single-Cell Multiomics, and Cardiac Organoids to Decode Genetic Variants
Grant Award Details
Grant Type:
Grant Number:
DISC0-18038
Investigator(s):
Award Value:
$4,606,248
Status:
Pre-Active
Grant Application Details
Application Title:
Base Editing, Single-Cell Multiomics, and Cardiac Organoids to Decode Genetic Variants
Public Abstract:
Research Objective
Develop a high-throughput platform combining iPSC-derived cardiac organoids and CRISPR base editing to functionally assess missense variants in hypertrophic cardiomyopathy.
Impact
(i) Limited tools for functional interpretation of missense variants, (ii) lack of scalable human cardiac models, and (iii) poor understanding of variant effects across genetic backgrounds.
Develop a high-throughput platform combining iPSC-derived cardiac organoids and CRISPR base editing to functionally assess missense variants in hypertrophic cardiomyopathy.
Impact
(i) Limited tools for functional interpretation of missense variants, (ii) lack of scalable human cardiac models, and (iii) poor understanding of variant effects across genetic backgrounds.