An innovative genetic therapy known as VERVE-101 has the potential to inactivate a gene that raises cholesterol levels in individuals with hypercholesterolemia, as reported by Meghan Rosen in the article “Base editing can lower cholesterol” (SN: 1/27/24, p. 8). However, insufficient dosage of VERVE-101 may hinder its effectiveness in turning off the gene in the liver, leading to inadequate cholesterol reduction. In such cases, a second dose of the drug may be necessary, although the developers advocate for a single-dose treatment.
Responding to a query from reader Jack Miller, Rosen clarified that VERVE-101 does not impact germ cells, which are responsible for producing sperm and egg cells. Studies in mice have shown that the drug primarily targets the liver and does not affect the germ line or the offspring of treated mice. Therefore, if the children of treated patients also have hypercholesterolemia, they would require their own treatment.
Source: www.sciencenews.org
Date: 2024-03-03 07:00:00