Single-course in vivo base editing therapy proven to lower cholesterol
Drug Discovery World
NOVEMBER 14, 2023
The trial participants have heterozygous familial hypercholesterolemia (HeFH), a life-threatening inherited disease characterised by lifelong elevations in blood LDL-C and accelerated atherosclerotic cardiovascular disease (ASCVD). Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments.
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