Creating 3D Retinas from Stem Cells to Study Early Blindness
Pluripotent Stem Cell Derived 3D Retinas for Studies of Early Onset Retinal Degeneration
['FUNDING_R01'] · UNIVERSITY OF CALIFORNIA, SAN DIEGO · NIH-10819505
This study is looking at a type of eye disease that causes blindness in kids, called Leber congenital amaurosis, and it uses special lab-grown eye cells to learn more about what goes wrong and how we might be able to fix it with new treatments.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CALIFORNIA, SAN DIEGO (nih funded) |
| Locations | 1 site (LA JOLLA, UNITED STATES) |
| Trial ID | NIH-10819505 on ClinicalTrials.gov |
What this research studies
This research focuses on understanding early onset retinal degeneration diseases, particularly Leber congenital amaurosis (LCA), which leads to blindness in children. By using human pluripotent stem cells to create 3D retinal models, researchers aim to investigate the mechanisms behind photoreceptor cell death. The study will explore specific gene mutations associated with these conditions to develop potential gene therapies. This innovative approach could provide insights into the disease process and pave the way for new treatments.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Leber congenital amaurosis or other inherited retinal degenerations.
Not a fit: Patients with retinal degeneration not linked to genetic mutations or those with advanced stages of the disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that prevent or reverse blindness caused by genetic retinal diseases.
How similar studies have performed: Previous research has shown promise in using stem cell-derived models to study retinal diseases, indicating that this approach could yield valuable insights.
Where this research is happening
LA JOLLA, UNITED STATES
- UNIVERSITY OF CALIFORNIA, SAN DIEGO — LA JOLLA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: WAHLIN, KARL J — UNIVERSITY OF CALIFORNIA, SAN DIEGO
- Study coordinator: WAHLIN, KARL J
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.