Verve Therapeutics’ next-generation gene editing treatment lowered cholesterol levels in a handful of patients with heterozygous familial hypercholesterolemia, marking the first time that base editing has been used to directly alter DNA in humans.
The early Phase I data, presented Sunday at the American Heart Association’s scientific meeting, give a glimpse at how the in vivo treatment works in people who have been diagnosed with the genetic disorder that causes high cholesterol and often heart disease at an early age.
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