Gene Editing for Familial Hypercholesterolemia
January 16, 2024 | Terry Sharrer
“In heterozygous familial hypercholesterolemia (HeFH), people inherit one gene that results in very high LDL-C levels and elevates the risk of heart attack at an early age. But interim results from an ongoing trial, the first in humans, have shown that a single infusion of a gene-editing treatment could significantly reduce LDL-C in people with HeFH, an effect that might be sustained for many years.” Verve Therapeutics, Boston, MA conducted the study. MORE
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