CRISPR-based exon-skipping to treat Usher syndrome deafness
Official title: Development of CRISPR/Cas9-based exon-skipping strategies for the treatment of USH-associated deafness
Funded at University of Miami School of Medicine · NIH-11125786 · Data updated 2026-11-12
A gene-editing approach using CRISPR techniques to fix faulty exon regions in Usher syndrome patients to protect hearing and balance functions.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Miami School of Medicine NIH-funded |
| Lab location | 1 site (Coral Gables, United States) |
| Project start | August 22, 2022 |
| Project end | June 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $666,228 |
| NIH project number | 5R01DC019404-04 |
| Project ID | NIH-11125786 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals with Usher syndrome or those carrying USH gene mutations who could provide patient-derived cells for organoid models or be considered for future trials.
✕Less likely to help
Patients without USH-related genetic mutations or those not eligible for gene-editing approaches may not benefit.
What this research studies
This project aims to develop genome-editing strategies using CRISPR to skip faulty exons in USH genes, tested first in human hair cells grown from patient-derived induced pluripotent stem cells and in USH mouse models. Researchers will assess safety and effectiveness of the edits in restoring normal gene function and improving cell health in the inner ear. The work includes creating organoid models of the human inner ear to study how edits affect hair cells and hearing-related biology. If successful, this work could lay the groundwork for future therapies that could help people with Usher syndrome maintain hearing and reduce progression of deaf-blindness.
Why it matters
Potential benefit: If successful, the therapy could preserve or restore hearing in people with Usher syndrome and slow progression toward blindness.
How similar studies have gone: Prior studies show promise for genome editing in hearing cells, but applying CRISPR to Usher-related genes in human inner-ear models is still early-stage.
Where this research is happening
-
University of Miami School of Medicine
Coral Gables, United States Active
Researchers
- Principal investigator: Blanton, Susan Halloran — University of Miami School of Medicine
- Study coordinator: Blanton, Susan Halloran
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.