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Heart Health
Gene editing: A one-time fix for dangerously high cholesterol?
This therapy — which disables a gene that regulates LDL cholesterol levels — is being tested in high-risk heart attack survivors. Here’s a look at the background and potential future of gene editing for heart disease.
- By Julie Corliss, Executive Editor, Harvard Heart Letter
- Reviewed by Christopher P. Cannon, MD, Editor in Chief, Harvard Heart Letter; Editorial Advisory Board Member, Harvard Health Publishing
Among your more than 20,000 different genes, one provides instructions for a protein called PCSK9, which helps regulate levels of "bad" LDL cholesterol in your bloodstream. About one in 250 people has a variant in this gene that causes LDL cholesterol to skyrocket, sometimes to more than three times the optimal ceiling of 100 mg/dL. This genetic condition, called familial hypercholesterolemia, is the leading cause of premature heart attacks in America.
But other people have different PCSK9 gene variants that have the exact opposite effect. Their LDL cholesterol is extraordinarily low—and their risk of heart attack is also far below average. This discovery, made by Dallas Heart Study researchers in 2005, spurred industry scientists to develop drugs called PCSK9 inhibitors (see "How does PCSK9 affect your cholesterol level?").
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About the Author
Julie Corliss, Executive Editor, Harvard Heart Letter
About the Reviewer
Christopher P. Cannon, MD, Editor in Chief, Harvard Heart Letter; Editorial Advisory Board Member, Harvard Health Publishing
Disclaimer:
As a service to our readers, Harvard Health Publishing provides access to our library of archived content. Please note the date of last review or update on all articles.
No content on this site, regardless of date, should ever be used as a substitute for direct medical advice from your doctor or other qualified clinician.
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Managing Your Cholesterol
Managing Your Cholesterol offers up-to-date information to help you or a loved one keep cholesterol in check. The report spells out what are healthy and unhealthy cholesterol levels, and offers specific ways to keep cholesterol in line. It covers cholesterol tests and the genetics of cholesterol. The report also focuses on treatments based on the latest scientific evidence, including the pros and cons of statins and other medications, and provides the lowdown on other substances advertised to lower cholesterol. Managing Your Cholesterol can also help you work with your doctor to individualize your treatment.
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