Join the club for FREE to access the whole archive and other member benefits.

Gene editing lowered blood level cholesterol and triglycerides in monkeys

Could prevent not only the complications but the heart disease per se in the future


Key points from article :

Scientists have disabled two genes in monkeys that raise the risk for heart disease.

One is PCSK9, which helps regulate levels of LDL cholesterol.

The other is ANGPTL3, part of the system regulating triglyceride, a type of blood fat.

Both are active in liver, which is where cholesterol and triglycerides are produced.

People who inherit mutations that destroyed the genes’ function do not get heart disease.

The medicine consists of two pieces of RNA — a gene editor and a tiny guide.

Not only did the system work in 13 monkeys, but it appeared that every liver cell was edited.

The monkeys’ LDL levels dropped by 59 percent within two weeks.

The ANGPTL3 gene editing led to a 64 percent decline in triglyceride levels.

First study to use the pencil-and-eraser type gene editing in primates for a very common disease.

But much too soon to say if it will be safe and long-lasting.

Research from Verve Therapeutics; not yet been peer-reviewed or published.

Mentioned in this article:

Click on resource name for more details.

Broad Institute of MIT and Harvard

Collaborative organization that brings academics of various disciplines in the scientific community together

Deepak Srivastava

Professor in Pediatrics

Gladstone Institutes

Nonprofit life science research organization

Jennifer Doudna

Professor of Biochemistry & Molecular Biology at UC Berkeley

Massachusetts General Hospital (MGH)

Largest teaching hospital of Harvard Medical School located in Boston

Michael Davidson

Professor, Director of the Lipid Clinic, The University of Chicago

Sekar Kathiresan

Physician scientist, human geneticist and professor at Harvard Medical School.

The University of Chicago

Public Research university.

University of California, Berkeley

Public land-grant research university

Verve Therapeutics

Biotechnology company

Topics mentioned on this page:
Heart Disease, Gene Therapy