Using gene editing to treat genetic hearing loss
Official title: AAV-mediated editing to treat human autosomal dominant hearing loss DFNA41 and DFNA2
Funded at Massachusetts Eye and Ear Infirmary · NIH-11041663 · Data updated 2026-11-12
This study is exploring new gene editing treatments for people with genetic hearing loss caused by specific conditions, aiming to safely fix the genetic issues that lead to hearing problems.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts Eye and Ear Infirmary NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 20, 2024 |
| Project end | August 31, 2028 |
| Funding year | 2024 |
| Award (this year) | $3,314,560 |
| NIH project number | 1U01TR005352-01 |
| Project ID | NIH-11041663 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with DFNA41 or DFNA2 genetic hearing loss, particularly those who are newborns or young children.
✕Less likely to help
Patients with hearing loss due to non-genetic factors or those with different genetic mutations not targeted by this research may not benefit.
What this research studies
This research aims to develop innovative gene editing therapies for genetic hearing loss, specifically targeting two conditions known as DFNA41 and DFNA2. The approach involves using a delivery method called AAV2 to introduce CRISPR-Cas-based therapeutics that can repair or disrupt the genetic mutations causing these hearing loss conditions. The research team consists of experts in gene therapy and will conduct studies to ensure the safety and effectiveness of these treatments in humans. By addressing the challenges of delivering gene editing tools effectively, this project hopes to pave the way for new treatments for patients suffering from genetic hearing loss.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for individuals with genetic hearing loss, potentially restoring their hearing.
How similar studies have gone: Previous research has shown success in gene therapy for other types of genetic hearing loss, indicating potential for this novel approach.
Where this research is happening
-
Massachusetts Eye and Ear Infirmary
Boston, United States Active
Researchers
- Principal investigator: Chen, Zheng-Yi — Massachusetts Eye and Ear Infirmary
- Study coordinator: Chen, Zheng-Yi
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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.