Gene therapy for a rare genetic disorder that causes rapid aging in children.
Gene Therapy in Hutchinson-Gilford Progeria Syndrome
This study is exploring a new way to fix the genetic problem that causes Hutchinson-Gilford Progeria Syndrome, which affects children, by using advanced techniques to help improve their health and lifespan.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Vanderbilt University Medical Center NIH-funded |
| Lab location | 1 site (Nashville, United States) |
| Project ID | NIH-11030823 on NIH RePORTER |
What this research studies
This research focuses on Hutchinson-Gilford Progeria Syndrome (HGPS), a severe genetic condition caused by a mutation in the Lamin A gene. The project aims to utilize advanced gene editing techniques to correct this mutation, potentially reversing the harmful effects of the disease. By targeting the genetic defect at its source, the researchers hope to improve the health and longevity of affected children. The study will involve testing this innovative approach in animal models before considering its application in humans.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with Hutchinson-Gilford Progeria Syndrome, typically under the age of 2.
Not a fit: Patients who do not have Hutchinson-Gilford Progeria Syndrome or are older than 21 years may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that significantly improves the quality of life and lifespan for children with HGPS.
How similar studies have performed: While gene editing is a relatively novel approach for treating genetic disorders, preliminary studies have shown promise in similar applications, indicating potential for success.
Where this research is happening
Nashville, United States
- Vanderbilt University Medical Center — Nashville, United States (Active)
Researchers
- Principal investigator: Brown, Jonathan David — Vanderbilt University Medical Center
- Study coordinator: Brown, Jonathan David
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.