Gene-editing for high cholesterol shows early promise.

Early-stage CRISPR studies that aim to switch off liver genes such as ANGPTL3 and PCSK9 have shown substantial LDL and triglyceride reductions in small groups of participants, but longer and larger trials are needed and safety questions remain.

Scientists are testing gene-editing as a possible one-time approach to lower high cholesterol in the liver. The work is very early and has been tried in only a few dozen people so far. The research builds on observations that people with naturally inactive ANGPTL3 or PCSK9 genes have very low LDL and triglyceride levels. Meanwhile, many people currently manage cholesterol with medicines such as statins and other injected or pill treatments. What is known: - Small clinical studies used CRISPR-based infusions to target genes in the liver, including ANGPTL3 and PCSK9, and reported rapid reductions in LDL cholesterol and triglycerides in some participants. - In one ANGPTL3 study of 15 adults, the highest dose group saw roughly a 50% drop in LDL and triglycerides within two weeks, the article reports. - Verve Therapeutics, a company mentioned in the article, reported a similar LDL reduction in a small PCSK9-directed study; the article says U.S. sites for follow-up studies are opening. - Gene edits in liver cells are expected to be long-lasting or permanent if edited cells reproduce, and some participants in earlier studies have shown sustained lowering for up to two years, the article notes. - Experts cited in the article warn that long-term safety is not yet known, and potential concerns include liver inflammation from delivery particles and the possibility of unintended edits to other genes. Summary: The reported results suggest gene-editing can substantially lower cholesterol measures in small, early trials, which has prompted plans for larger follow-up studies. Key open questions include long-term safety, durability in larger populations, and precise targeting; next-step studies are planned but broader outcomes remain undetermined at this time.