Fixing Genes for Familial High Cholesterol
Genetic Repair of Familial Hypercholesterolemia
['FUNDING_R01'] · BAYLOR COLLEGE OF MEDICINE · NIH-11059209
This project is developing new ways to fix the genes that cause a severe form of very high cholesterol called Familial Hypercholesterolemia.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | BAYLOR COLLEGE OF MEDICINE (nih funded) |
| Locations | 1 site (HOUSTON, UNITED STATES) |
| Trial ID | NIH-11059209 on ClinicalTrials.gov |
What this research studies
Familial Hypercholesterolemia (FH) is a genetic condition that causes extremely high cholesterol, and a severe type called homozygous FH (HoFH) can be life-threatening without treatment. Currently, a liver transplant is the only cure for HoFH, which highlights the need for better options. Researchers are working on gene editing techniques to permanently correct the faulty DNA in liver cells responsible for this condition. They are testing strategies to insert a healthy gene into safe spots within the DNA and using mouse models to explore how gene-corrected liver cells can grow and replace unhealthy ones. The goal is to create a therapy that can help many HoFH patients, regardless of their specific genetic mutation.
Who could benefit from this research
Good fit: This research is most relevant for individuals diagnosed with homozygous Familial Hypercholesterolemia (HoFH), a severe genetic form of high cholesterol.
Not a fit: Patients with high cholesterol due to lifestyle factors or other genetic causes not related to LDLR mutations may not directly benefit from this specific gene therapy approach.
Why it matters
Potential benefit: If successful, this work could lead to a permanent cure for severe familial high cholesterol, potentially preventing serious health issues and the need for liver transplants.
How similar studies have performed: While gene editing is a promising and evolving field, a permanent gene therapy cure for homozygous Familial Hypercholesterolemia is still in the developmental stages and has not yet been established.
Where this research is happening
HOUSTON, UNITED STATES
- BAYLOR COLLEGE OF MEDICINE — HOUSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LAGOR, WILLIAM RAYMOND — BAYLOR COLLEGE OF MEDICINE
- Study coordinator: LAGOR, WILLIAM RAYMOND
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.