Deep phenotyping of human brain organoid models of autism spectrum disorder to unravel disease heterogeneity and develop biomarkers and treatments
Grant Award Details
Grant Type:
Grant Number:
DISC4-16285
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Cell Line Generation:
Award Value:
$12,297,272
Status:
Pre-Active
Grant Application Details
Application Title:
Deep phenotyping of human brain organoid models of autism spectrum disorder to unravel disease heterogeneity and develop biomarkers and treatments
Public Abstract:
Research Objective
We will uncover pathways through which ASD mutations cause disease and close the gap from disease research to therapeutic testing using organoids, primary human neurons, machine learning and AAVs.
Impact
Our studies are impactful because outcomes will lead to therapeutic avenues to pursue for ASD treatment.
We will uncover pathways through which ASD mutations cause disease and close the gap from disease research to therapeutic testing using organoids, primary human neurons, machine learning and AAVs.
Impact
Our studies are impactful because outcomes will lead to therapeutic avenues to pursue for ASD treatment.
Statement of Benefit to California:
1 in 22 children in California is diagnosed with autism, up from a previous estimates of 1 in 44 children in December 2022. Our study will identfiy the pathways through which ASD genetic mutations cause disease and will uncover new therapeutic targets. With our interdisciplinary team, we are able to close the gap from disease research to therapeutic testing using the most human relevant disease models, machine learning, CRISPR screening and AAVs.