Award
NIH Reporter #5R01DC019404-04
Development of CRISPR/Cas9-based exon-skipping strategies for the treatment of USH-associated deafness
Recipient
UNIVERSITY OF MIAMI SCHOOL OF MEDICINE
Award Amount
$666,228.00
Ceiling
$666,228.00
Awarded
August 28, 2026
Identifier
5R01DC019404-04
This award supports the development of CRISPR/Cas9 exon-skipping therapies for USH-associated deafness, using human inner ear organoids and mouse models to target specific USH genes.
Description
The project aims to develop and test genome editing-based therapeutic strategies for USH-associated deafness, focusing on USH2A, USH1D, and USH1F genes. It involves creating human inner ear organoids from hiPSCs and testing exon-skipping strategies in mouse models. The project is funded by NIH grant PA-20-185.