AAV-based gene editing to treat inherited hearing loss (DFNA41 and DFNA2)
Official title: AAV-mediated editing to treat human autosomal dominant hearing loss DFNA41 and DFNA2
Funded at Massachusetts Eye and Ear Infirmary · NIH-11384748 · Data updated 2026-11-12
A gene-editing therapy delivered by AAV aims to treat certain inherited forms of hearing loss in adults and children.
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 | 2026 |
| Award (this year) | $4,610,714 |
| NIH project number | 5U01TR005352-03 |
| Project ID | NIH-11384748 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults and children with DFNA41 (P2RX2) or DFNA2 (KCNQ4)–related hearing loss who are eligible for gene therapy trials.
✕Less likely to help
People with hearing loss due to causes other than DFNA41 or DFNA2 may not benefit from this approach.
What this research studies
This project focuses on delivering a CRISPR-based therapy to the inner ear using AAV vectors to target dominant mutations causing DFNA41 and DFNA2 hearing loss. It plans to move from lab studies toward IND-enabling steps, with a team of experts in gene editing, delivery methods, and regulatory pathways. The goal is to show safe and effective delivery to human ear cells and pave the way for a future clinical trial. If successful, the approach could offer a one-time treatment option for people with these specific genetic hearing losses.
Why it matters
Potential benefit: If successful, this therapy could restore or preserve hearing in people with these genetic conditions.
How similar studies have gone: Preclinical gene therapies for hearing loss have shown promise; an earlier successful AAV-based approach in related conditions informs this work, but this exact therapy is building on novel targets and delivery.
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.