A new gel for delivering gene therapy to the retina
Retinal-adhesive thermoresponsive gel for AAV-mediated gene delivery to the outer retina
['FUNDING_R01'] · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · NIH-10898763
This study is working on a special gel that can help deliver gene therapy directly to the outer part of the retina, aiming to treat retinal diseases while protecting healthy cells, so people with vision problems can have better treatment options.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF PITTSBURGH AT PITTSBURGH (nih funded) |
| Locations | 1 site (PITTSBURGH, UNITED STATES) |
| Trial ID | NIH-10898763 on ClinicalTrials.gov |
What this research studies
This research focuses on developing a thermoresponsive gel that can effectively deliver gene therapy to the outer retina, specifically targeting retinal degenerations. The approach aims to improve the delivery of adeno-associated viruses (AAV) to affected cells while minimizing damage to healthy photoreceptors. By addressing challenges such as efficient targeting and reducing inflammatory responses, this research seeks to enhance the effectiveness of gene therapies for various retinal diseases.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals suffering from retinal degenerations, particularly those with conditions that may benefit from gene therapy.
Not a fit: Patients with retinal conditions that are not amenable to gene therapy or those who do not have structural alterations in their retina may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a more effective treatment option for patients with retinal degenerations, potentially restoring vision.
How similar studies have performed: Previous research has shown promising results with gene therapy approaches for retinal diseases, indicating a potential for success with this novel delivery method.
Where this research is happening
PITTSBURGH, UNITED STATES
- UNIVERSITY OF PITTSBURGH AT PITTSBURGH — PITTSBURGH, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: SAHEL, JOSE-ALAIN — UNIVERSITY OF PITTSBURGH AT PITTSBURGH
- Study coordinator: SAHEL, JOSE-ALAIN
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.