New treatments for inherited deaf-blindness caused by Usher syndrome
Official title: Development of CRISPR/Cas9-based exon-skipping strategies for the treatment of USH-associated deafness
Funded at University of Miami School of Medicine · NIH-10876488 · Data updated 2026-11-12
This study is exploring new ways to help people with Usher syndrome, which causes both hearing and vision loss, by using a special technology to fix the genes that are causing these issues, and patients might even get a chance to be part of this exciting research that could lead to better treatments.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Miami School of Medicine NIH-funded |
| Lab location | 1 site (Coral Gables, United States) |
| Project start | August 22, 2022 |
| Project end | June 30, 2027 |
| Funding year | 2024 |
| Award (this year) | $689,919 |
| NIH project number | 5R01DC019404-03 |
| Project ID | NIH-10876488 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Usher syndrome, particularly those with mutations in the USH2A, USH1D, or USH1F genes.
✕Less likely to help
Patients with Usher syndrome who do not have the specific genetic mutations targeted by this research may not benefit from the outcomes.
What this research studies
This research focuses on developing innovative therapies for Usher syndrome, the most common inherited cause of deaf-blindness. By utilizing CRISPR/Cas9 technology, the project aims to create strategies that can skip faulty gene segments in patients' cells, potentially restoring hearing and vision. The research involves testing these approaches in human stem cell-derived inner ear organoids and mouse models to evaluate their effectiveness. Patients may have the opportunity to contribute to this groundbreaking work and potentially benefit from new treatment options.
Why it matters
Potential benefit: If successful, this research could lead to effective therapies that improve hearing and vision for individuals with Usher syndrome.
How similar studies have gone: While the use of CRISPR technology in gene therapy is a promising area, this specific approach for treating Usher syndrome is novel and has not been extensively tested in clinical settings.
Where this research is happening
-
University of Miami School of Medicine
Coral Gables, United States Active
Researchers
- Principal investigator: Liu, Xue Z — University of Miami School of Medicine
- Study coordinator: Liu, Xue Z
Related research
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This grant studies how to improve speech understanding and spatial hearing for people using cochlear implants, especially those with implants in both ears or with single-sided deafness.
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This project works to make genetic test results clearer and more useful for people with inherited hearing loss.
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How Usher-1 proteins in inner-ear hair cells affect hearing
This project looks at how key Usher-1 proteins help tiny hair cells in the inner ear work and how problems in these proteins lead to inherited hearing and vision loss for people with Usher syndrome.
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.