Treatment for vision loss in Usher syndrome using antisense therapy
Antisense therapy for the treatment of visual loss in Usher syndrome
This study is exploring a new treatment for vision loss caused by Usher syndrome, which also affects hearing, and it aims to help patients like you by testing a special therapy that targets the genetic issues behind the condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Lsu Health Sciences Center NIH-funded |
| Lab location | 1 site (New Orleans, United States) |
| Project ID | NIH-10661603 on NIH RePORTER |
What this research studies
This research focuses on developing a new treatment for vision loss associated with Usher syndrome, a rare genetic disorder that also causes hearing loss. The approach involves using antisense oligonucleotides (ASOs) to target specific genetic mutations responsible for the condition. By identifying measurable clinical parameters, the research aims to establish effective endpoints for future clinical trials. Patients may have the opportunity to participate in trials that assess the safety and efficacy of this innovative therapy.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Usher syndrome, particularly those with the USH1C gene mutation.
Not a fit: Patients with Usher syndrome who do not have the specific genetic mutation targeted by the antisense therapy may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could significantly improve vision and quality of life for patients with Usher syndrome.
How similar studies have performed: Other research has shown promise in using antisense therapy for genetic disorders, indicating potential for success in this novel approach.
Where this research is happening
New Orleans, United States
- Lsu Health Sciences Center — New Orleans, United States (Active)
Researchers
- Principal investigator: Lentz, Jennifer Jean — Lsu Health Sciences Center
- Study coordinator: Lentz, Jennifer Jean
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.