By Gina Kolata June 27, 2020
What if a single injection could lower blood levels of cholesterol and triglycerides — for a lifetime?
In the first gene-editing experiment of its kind, scientists have disabled two genes in monkeys that raise the risk for heart disease. Humans carry the genes as well, and the experiment has raised hopes that a leading killer may one day be tamed.
“This could be the cure for heart disease,” said Dr. Michael Davidson, director of the Lipid Clinic at the University of Chicago Pritzker School of Medicine, who was not involved in the research.
But it will be years before human trials can begin, and gene-editing technology so far has a mixed tracked record. It is much too early to know whether the strategy will be safe and effective in humans; even the monkeys must be monitored for side effects or other treatment failures for some time to come.
The results were presented on Saturday at the annual meeting of the International Society for Stem Cell Research, this year held virtually with about 3,700 attendees around the world. The scientists are writing up their findings, which have not yet been peer-reviewed or published.
What if a single injection could lower blood levels of cholesterol and triglycerides — for a lifetime?
In the first gene-editing experiment of its kind, scientists have disabled two genes in monkeys that raise the risk for heart disease. Humans carry the genes as well, and the experiment has raised hopes that a leading killer may one day be tamed.
“This could be the cure for heart disease,” said Dr. Michael Davidson, director of the Lipid Clinic at the University of Chicago Pritzker School of Medicine, who was not involved in the research.
But it will be years before human trials can begin, and gene-editing technology so far has a mixed tracked record. It is much too early to know whether the strategy will be safe and effective in humans; even the monkeys must be monitored for side effects or other treatment failures for some time to come.
The results were presented on Saturday at the annual meeting of the International Society for Stem Cell Research, this year held virtually with about 3,700 attendees around the world. The scientists are writing up their findings, which have not yet been peer-reviewed or published.
The researchers set out to block two genes: PCSK9, which helps regulate levels of LDL cholesterol; and ANGPTL3, part of the system regulating triglyceride, a type of blood fat. Both genes are active in the liver, which is where cholesterol and triglycerides are produced. People who inherit mutations that destroyed the genes’ function do not get heart disease.
People with increased blood levels of triglycerides and LDL cholesterol have dramatically greater risks of heart disease, heart attacks and strokes, the leading causes of death in most of the developed world. Drug companies already have developed and are marketing two so-called PCSK9 inhibitors that markedly lower LDL cholesterol, but they are expensive and must be injected every few weeks.
Researchers at Verve Therapeutics, led by Dr. Sekar Kathiresan, the chief executive, decided to edit the genes instead. The medicine they developed consists of two pieces of RNA — a gene editor and a tiny guide that directs the editor to a single sequence of 23 letters of human DNA among the genome’s 32.5 billion letters.