An experimental therapy with CRISPR-Cas9 technology has significantly reduced LDL cholesterol and triglycerides, even a year after a single dose. The researchers consider the result promising, but warn that larger trials are needed.

A new genetic treatment could open a new avenue for patients with very high and difficult-to-control cholesterol.

The experimental therapy CTX310 targets the ANGPTL3 gene in the liver, which is related to the regulation of cholesterol and triglycerides. In the first phase study, with only 15 patients, the highest dose of treatment lowered LDL by about 53% even after one year, while triglycerides decreased by nearly 48%.

The result is particularly interesting because it is a single dose, rather than treatments that must be taken continuously.

However, the scientists emphasize that these are very early results. CRISPR directly alters DNA, and the effect can be irreversible, so long-term safety remains a major concern.

The study also reported about a 20% drop in HDL, while one patient who received the lowest dose died 179 days after treatment; the authors did not link the death to the therapy.

If the results are confirmed in larger studies, this technology could change the way high cholesterol is treated. For now, however, CTX310 remains an experimental therapy and does not replace existing treatments.

Source: prizrenpost