New treatments for inherited deaf-blindness caused by Usher syndrome
Development of CRISPR/Cas9-based exon-skipping strategies for the treatment of USH-associated deafness
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 ID | NIH-10876488 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Usher syndrome, particularly those with mutations in the USH2A, USH1D, or USH1F genes.
Not a fit: Patients with Usher syndrome who do not have the specific genetic mutations targeted by this research may not benefit from the outcomes.
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 performed: 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
Coral Gables, United States
- 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
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.