Gene editing lowered blood level cholesterol and triglycerides in monkeys
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Research from Verve Therapeutics; not yet been peer-reviewed or published.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:
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 Biomedical Science
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.
Verve Therapeutics - Biotechnology company