Targeting genetic causes of hearing loss and balance dysfunction
Official title: Genetic Approach to Therapy for DFNA9
Funded at Brigham and Women's Hospital · NIH-11063134 · Data updated 2026-11-12
This study is working on new treatments for DFNA9, a genetic condition that causes hearing loss and balance problems, by using a special gene-editing tool to fix the harmful mutations in the COCH gene, with the hope of helping those affected by this condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brigham and Women's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | April 20, 2023 |
| Project end | March 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $703,055 |
| NIH project number | 5R01DC021104-03 |
| Project ID | NIH-11063134 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with DFNA9 or those carrying the specific COCH gene mutations.
✕Less likely to help
Patients with hearing loss not related to the COCH gene mutations or those with other unrelated balance disorders may not benefit from this research.
What this research studies
This research focuses on developing targeted therapies for DFNA9, a genetic condition that leads to hearing loss and balance issues. Using advanced CRISPR-Cas9 gene editing technology, the team aims to specifically disrupt harmful mutations in the COCH gene responsible for this disorder. By utilizing patient-derived cells, the researchers will create models to test and refine these therapeutic approaches, potentially leading to effective treatments for individuals affected by DFNA9.
Why it matters
Potential benefit: If successful, this research could provide new therapeutic options for patients suffering from genetic hearing loss and balance disorders.
How similar studies have gone: Previous research using CRISPR technology has shown promise in addressing genetic disorders, suggesting potential success for this novel approach.
Where this research is happening
-
Brigham and Women's Hospital
Boston, United States Active
Researchers
- Principal investigator: Morton, Cynthia Casson — Brigham and Women's Hospital
- Study coordinator: Morton, Cynthia Casson
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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.