Article

Base Editing Gene Therapy for Heterozygous Familial Hypercholesterolemia

2025-04-25

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Background</h4> Heterozygous familial hypercholesterolemia (HeFH) is a genetic disorder characterized by persistently elevated low-density lipoprotein cholesterol (LDL-C) levels, leading to an increased risk of early-onset atherosclerosis cardiovascular diseases (ASCVD). YOLT-101, an in vivo base-editing therapeutic agent delivered via GalNAc-modified lipid nanoparticles, is designed to ach...

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Literature Corpus work: 57685023-df42-5546-870d-3a6d7c50c50b
DOI: 10.1101/2025.04.17.25325983

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