This article reviews a gene therapy that prevents clogged arteries in people who have a genetic basis for high levels of low density lipoprotein cholesterol.
Later this year, Verve Therapeutics of Cambridge, Ma., will initiate Phase 1 clinical trials to test VERVE-101, a new medication that, if successful, will employ gene editing to significantly reduce low-density lipoprotein cholesterol, or LDL.
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The medication is targeted specifically for patients who have a genetic form of high cholesterol known as heterozygous familial hypercholesterolemia, or FH, caused by expression of a gene called PCSK9. Verve also plans to develop a program to silence a gene called ANGPTL3 for patients with FH and possibly those with or at risk of atherosclerotic cardiovascular disease.
They’ve done the testing in monkeys and soon they’ll be trying it out on humans. This is getting closer to the stuff that was science fiction fifty years ago. A single intravenous treatment alters the DNA sequence of the patient’s liver cells and permanently cures a condition that would otherwise require statins and dieting and eventually stents and surgery to keep the patient alive.
I’m waiting for the gene therapy to cure my congenital laziness. But the work-ethic-impaired never get consideration for our disease. So unfair.