Correcting a genetic mutation in blood vessels affected by Hutchinson-Gilford Progeria Syndrome
Investigating the Effects of Hutchinson-Gilford Progeria Syndrome Mutation Correction in Tissue-Engineered Blood Vessels
['FUNDING_FELLOWSHIP'] · DUKE UNIVERSITY · NIH-10996997
This study is looking at how a rare condition called Hutchinson-Gilford Progeria Syndrome affects blood vessels and aims to find ways to fix the genetic mutation that causes it, which could help improve heart health for those living with this condition.
Quick facts
| Phase | ['FUNDING_FELLOWSHIP'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | DUKE UNIVERSITY (nih funded) |
| Locations | 1 site (DURHAM, UNITED STATES) |
| Trial ID | NIH-10996997 on ClinicalTrials.gov |
What this research studies
This research focuses on Hutchinson-Gilford Progeria Syndrome (HGPS), a rare condition caused by a mutation in the LMNA gene that leads to accelerated aging and severe cardiovascular issues. The study aims to understand how different cell types in blood vessels contribute to the disease's progression, particularly atherosclerosis. By using advanced genetic engineering techniques, the researchers will explore the potential of correcting the mutation to improve vascular health. This could lead to more effective treatments for patients suffering from HGPS and related cardiovascular complications.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Hutchinson-Gilford Progeria Syndrome.
Not a fit: Patients with other forms of accelerated aging or unrelated cardiovascular diseases may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to improved therapies that significantly enhance the quality of life and longevity for patients with Hutchinson-Gilford Progeria Syndrome.
How similar studies have performed: Previous research using genetic engineering approaches has shown promise in animal models, but this specific application in humans remains novel.
Where this research is happening
DURHAM, UNITED STATES
- DUKE UNIVERSITY — DURHAM, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: SHORES, KEVIN — DUKE UNIVERSITY
- Study coordinator: SHORES, KEVIN
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.
Conditions: Atherosclerotic Cardiovascular Disease