Understanding cholesterol balance in the retina
Cholesterol homeostasis in the vertebrate retina
['FUNDING_R01'] · STATE UNIVERSITY OF NEW YORK AT BUFFALO · NIH-11043384
This study is looking at how cholesterol levels in the eye are kept healthy, especially for people with genetic conditions like Smith-Lemli-Opitz Syndrome that can cause serious vision issues, and it uses special mouse models to learn how retinal cells can still work even when they don't have enough cholesterol.
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-11043384 on ClinicalTrials.gov |
What this research studies
This research investigates how cholesterol levels are maintained in the retina, which is crucial for the health of retinal cells. It focuses on hereditary conditions like Smith-Lemli-Opitz Syndrome that disrupt cholesterol synthesis, leading to severe vision problems. The researchers have developed new mouse models to study the effects of cholesterol deficiency on retinal cells and aim to understand how these cells can survive and function despite these deficiencies. By examining the role of cholesterol uptake from blood and other cells in the retina, the study seeks to uncover potential therapeutic targets.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals with hereditary cholesterol synthesis disorders, particularly those affected by Smith-Lemli-Opitz Syndrome.
Not a fit: Patients with retinal diseases not related to cholesterol metabolism may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments for retinal diseases caused by cholesterol imbalances.
How similar studies have performed: While the specific approach of using conditional allele models for studying cholesterol homeostasis in the retina is novel, related research has shown promise in understanding cholesterol's role in other tissues.
Where this research is happening
AMHERST, UNITED STATES
- STATE UNIVERSITY OF NEW YORK AT BUFFALO — AMHERST, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: FLIESLER, STEVEN J. — STATE UNIVERSITY OF NEW YORK AT BUFFALO
- Study coordinator: FLIESLER, STEVEN 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.