Targeting genetic causes of hearing loss and balance dysfunction
Genetic Approach to Therapy for DFNA9
['FUNDING_R01'] · BRIGHAM AND WOMEN'S HOSPITAL · NIH-11063134
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
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | BRIGHAM AND WOMEN'S HOSPITAL (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-11063134 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with DFNA9 or those carrying the specific COCH gene mutations.
Not a fit: Patients with hearing loss not related to the COCH gene mutations or those with other unrelated balance disorders may not benefit from this research.
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 performed: Previous research using CRISPR technology has shown promise in addressing genetic disorders, suggesting potential success for this novel approach.
Where this research is happening
BOSTON, UNITED STATES
- 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
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 below.
Conditions: balance disorder