Fixing Genes for Familial High Cholesterol
Official title: Genetic Repair of Familial Hypercholesterolemia
Funded at Baylor College of Medicine · NIH-11059209 · Data updated 2026-11-12
This project is developing new ways to fix the genes that cause a severe form of very high cholesterol called Familial Hypercholesterolemia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Baylor College of Medicine NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project start | December 20, 2016 |
| Project end | March 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $558,477 |
| NIH project number | 5R01HL132840-09 |
| Project ID | NIH-11059209 on NIH RePORTER |
Who could benefit from this research
✓Could help
This research is most relevant for individuals diagnosed with homozygous Familial Hypercholesterolemia (HoFH), a severe genetic form of high cholesterol.
✕Less likely to help
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.
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.
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 gone: 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
-
Baylor College of Medicine
Houston, United States Active
Researchers
- Principal investigator: Lagor, William Raymond — Baylor College of Medicine
- Study coordinator: Lagor, William Raymond
Related research
Other NIH-funded projects on the same topics.
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Using behavioral nudges to improve family screening for inherited high cholesterol
This project uses two patient-facing approaches based on behavioral economics to help people with familial hypercholesterolemia get their relatives screened for the condition.
-
Improving Risk Stratification for Familial Hypercholesterolemia (FH)
A patient-facing look at a study that aims to better predict who with familial hypercholesterolemia is at highest risk for early heart disease.
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Nonviral CRISPR-Cas9 delivery to liver cells to treat familial hypercholesterolemia
A new approach uses nonviral methods to deliver CRISPR-Cas9 to liver cells and select edited cells with acetaminophen, aiming to permanently lower bad cholesterol in people with familial hypercholesterolemia.
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Implementing multi-component approaches to help blood donors with familial hypercholesterolemia(
A program to help people who donate blood and have familial hypercholesterolemia get the cholesterol-lowering treatment they need through a blood-donation center.
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Helping families find and treat inherited high cholesterol
This project tests a clinic-based program to help primary care doctors find and treat children and adults with inherited high cholesterol (familial hypercholesterolemia).
-
Familial hypercholesterolemia: closing gaps in prevention of heart disease
A new NIH grant is exploring why people with familial hypercholesterolemia often go undiagnosed or receive uneven care, with the goal of improving early detection and prevention of heart disease.
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.