Fixing common inherited hearing loss from GJB2 mutations
Gene Therapy for Hearing and Balance Disorders
This project develops gene-editing methods to correct the most common GJB2 mutations that cause inherited hearing loss for adults with DFNB1.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Harvard Medical School NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11306125 on NIH RePORTER |
What this research studies
Researchers are developing three gene-editing approaches—base editing, prime editing, and long-range prime editing—to correct the most common GJB2 mutations that cause DFNB1. They plan to use AAV viral delivery because the GJB2 gene is small enough to fit into a single vector, while also tackling durability issues since supporting cochlear cells divide and can dilute AAV episomes. The work uses cell-based tests and animal models and is carried out by collaborating labs at Harvard to refine the editors and delivery before any human testing. If the approaches are safe and effective in preclinical work, they could progress toward clinical trials for people with GJB2-related hearing loss.
Who could benefit from this research
Good fit: Ideal candidates would be adults (21+) with DFNB1 hereditary hearing loss caused by common GJB2 mutations such as c.35delG or p.M34T.
Not a fit: People whose hearing loss is caused by other genes, acquired/environmental damage, or who have severe, long-standing cochlear destruction may not benefit from these gene-editing approaches.
Why it matters
Potential benefit: If successful, these gene edits could provide a long-lasting correction of the genetic defect and restore more natural hearing for people with GJB2-related deafness.
How similar studies have performed: Preclinical gene therapies for hearing loss have shown promise in animal models, but applying base and prime editors in the ear is relatively new with limited human testing to date.
Where this research is happening
Boston, United States
- Harvard Medical School — Boston, United States (Active)
Researchers
- Principal investigator: Corey, David P — Harvard Medical School
- Study coordinator: Corey, David P
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.