Modeling Rett syndrome neurological disorder with human pluripotent stem cells to develop in cellulo screening platforms.
Grant Award Details
Grant Type:
Grant Number:
DISC0-17276
Investigator(s):
Award Value:
$2,393,281
Status:
Pre-Active
Grant Application Details
Application Title:
Modeling Rett syndrome neurological disorder with human pluripotent stem cells to develop in cellulo screening platforms.
Public Abstract:
Research Objective
Study the neurological disorder Rett syndrome using neurons derived from genetically engineered human pluripotent stem cells and develop platforms to screen drugs for this disease in live cells.
Impact
Rett syndrome is the second most common cause of girls’ intellectual disability, still lacking effective treatment. We hope to accelerate the discovery of more effective drugs for this orphan disease.
Study the neurological disorder Rett syndrome using neurons derived from genetically engineered human pluripotent stem cells and develop platforms to screen drugs for this disease in live cells.
Impact
Rett syndrome is the second most common cause of girls’ intellectual disability, still lacking effective treatment. We hope to accelerate the discovery of more effective drugs for this orphan disease.
