Improving treatments for inherited retinal diseases using advanced ribozymes
Optimizing Enhanced Hammerhead Ribozymes for Retinal Nucleic Acid Therapeutics
['FUNDING_R01'] · STATE UNIVERSITY OF NEW YORK AT BUFFALO · NIH-11086150
This study is testing a new eye treatment that uses a special type of gene therapy to help people with genetic eye conditions like retinitis pigmentosa by targeting and fixing the faulty genes without using viruses.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | STATE UNIVERSITY OF NEW YORK AT BUFFALO (nih funded) |
| Locations | 1 site (AMHERST, UNITED STATES) |
| Trial ID | NIH-11086150 on ClinicalTrials.gov |
What this research studies
This research focuses on developing a new type of gene therapy using Enhanced Hammerhead Ribozymes (EhhRz) to target mutations in genes responsible for autosomal dominant retinitis pigmentosa and other retinal degenerations. The approach aims to effectively reduce harmful mutant mRNA while restoring normal protein function without the need for viral vectors. By leveraging the unique properties of EhhRz, the researchers hope to create injectable therapies that can be used directly in the eye, potentially offering a new treatment option for patients with these genetic conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with autosomal dominant retinitis pigmentosa or other related retinal degenerations caused by specific genetic mutations.
Not a fit: Patients with retinal diseases not caused by mutations in the targeted genes may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a novel therapeutic option for patients suffering from inherited retinal diseases, potentially improving vision and quality of life.
How similar studies have performed: Recent clinical successes with antisense agents suggest that this approach has the potential for meaningful advancements in treating similar conditions.
Where this research is happening
AMHERST, UNITED STATES
- STATE UNIVERSITY OF NEW YORK AT BUFFALO — AMHERST, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: SULLIVAN, JOHN M. — STATE UNIVERSITY OF NEW YORK AT BUFFALO
- Study coordinator: SULLIVAN, JOHN M.
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.