Investigating treatments for corneal endothelial dystrophies
Analysis of chemical modulators for corneal endothelial dystrophies
['FUNDING_R01'] · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · NIH-10906819
This study is looking for new ways to help people with corneal endothelial dystrophies, a condition that can cause vision loss, by finding small molecules that can fix misfolded proteins caused by a specific gene mutation, offering hope for better treatments beyond just corneal transplants.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CALIFORNIA, SAN FRANCISCO (nih funded) |
| Locations | 1 site (SAN FRANCISCO, UNITED STATES) |
| Trial ID | NIH-10906819 on ClinicalTrials.gov |
What this research studies
This research focuses on corneal endothelial dystrophies, which lead to vision loss due to dysfunction of the corneal endothelium. The team is exploring how to correct misfolded proteins caused by mutations in the SLC4A11 gene, which is crucial for maintaining corneal health. By screening small molecules that can assist in proper protein folding, the researchers aim to develop new therapeutic options beyond corneal transplantation. This approach could potentially restore vision for patients suffering from these conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with corneal endothelial dystrophies, particularly those with mutations in the SLC4A11 gene.
Not a fit: Patients with corneal endothelial dystrophies not associated with SLC4A11 mutations may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to innovative treatments that restore vision for patients with corneal endothelial dystrophies.
How similar studies have performed: Previous research has shown promise in using small molecules to correct protein misfolding, indicating potential success for this approach.
Where this research is happening
SAN FRANCISCO, UNITED STATES
- UNIVERSITY OF CALIFORNIA, SAN FRANCISCO — SAN FRANCISCO, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: CHAN, MATILDA F — UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
- Study coordinator: CHAN, MATILDA F
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.