Gene editing therapy for a genetic disorder causing hearing and vision loss
Official title: Genome Editing Therapy for Usher Syndrome Type 3
Funded at Genetobe INC. · NIH-10759804 · Data updated 2026-11-12
This study is testing a new gene editing treatment for people with Usher syndrome type 3A, which causes hearing and vision loss, to see if it can safely fix the faulty gene and help improve their hearing and vision.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Genetobe INC. NIH-funded |
| Lab location | 1 site (Ann Arbor, United States) |
| Project start | September 1, 2023 |
| Project end | August 31, 2025 |
| Funding year | 2023 |
| Award (this year) | $289,895 |
| NIH project number | 1R42EY035582-01 |
| Project ID | NIH-10759804 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Usher syndrome type 3A who are experiencing progressive hearing and vision loss.
✕Less likely to help
Patients with other forms of Usher syndrome or those whose hearing and vision loss are not related to the CLRN1 gene mutation may not benefit from this research.
What this research studies
This research focuses on developing a gene editing therapy for Usher syndrome type 3A, a condition that leads to progressive hearing and vision loss due to mutations in the CLRN1 gene. The approach involves using a novel gene editing tool, meticulous integration Cas9 (miCas9), which aims to directly repair the defective gene in patients' genomes. By optimizing the delivery method and ensuring precise gene editing, the research seeks to provide a safer and more effective treatment option for individuals suffering from this genetic disorder. Patients may benefit from improved hearing and vision outcomes if the therapy proves successful.
Why it matters
Potential benefit: If successful, this research could restore or significantly improve hearing and vision in patients with Usher syndrome type 3A.
How similar studies have gone: Previous research has shown promise with gene editing approaches in animal models, but this specific application for Usher syndrome type 3A is novel and untested in humans.
Where this research is happening
-
Genetobe INC.
Ann Arbor, United States Active
Researchers
- Principal investigator: Yang, Dongshan — Genetobe INC.
- Study coordinator: Yang, Dongshan
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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.