Mechanisms underlying dosage sensitivity in developmental disorders
Grant Award Details
Grant Type:
Grant Number:
DISC0-17487
Investigator(s):
Award Value:
$2,304,000
Status:
Pre-Active
Grant Application Details
Application Title:
Mechanisms underlying dosage sensitivity in developmental disorders
Public Abstract:
Research Objective
This proposal leverages pluripotent stem cell models to understand the principles by which changes in gene and protein dosage affect human neural and facial progenitor development and cause disease.
Impact
Proposed research will illuminate mechanisms underlying neurodevelopmental and craniofacial diseases. It will also provide a framework for modeling dosage effects for improved gene and cell therapy.
This proposal leverages pluripotent stem cell models to understand the principles by which changes in gene and protein dosage affect human neural and facial progenitor development and cause disease.
Impact
Proposed research will illuminate mechanisms underlying neurodevelopmental and craniofacial diseases. It will also provide a framework for modeling dosage effects for improved gene and cell therapy.