A novel platform to rescue neurodevelopmental disorders caused by haploinsufficiency
Grant Award Details
Grant Type:
Grant Number:
DISC0-17488
Investigator(s):
Award Value:
$4,086,486
Status:
Pre-Active
Grant Application Details
Application Title:
A novel platform to rescue neurodevelopmental disorders caused by haploinsufficiency
Public Abstract:
Research Objective
We develop a powerful platform to correct gene expression defects caused by haploinsufficiency and will show its ability to identify novel targets using iPSC-derived neurons and cortical organoids.
Impact
De-repressing haploinsufficient genes from endogenous regulation has lacked high-throughput and resolution tools. Our technical innovations overcome this bottleneck enabling a new discovery platform.
We develop a powerful platform to correct gene expression defects caused by haploinsufficiency and will show its ability to identify novel targets using iPSC-derived neurons and cortical organoids.
Impact
De-repressing haploinsufficient genes from endogenous regulation has lacked high-throughput and resolution tools. Our technical innovations overcome this bottleneck enabling a new discovery platform.
