familial hypercholesterolemia — Hawaii Medical Journal

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In Vivo Base Editing for Familial Hypercholesterolemia: Early Evidence from a Phase 1 Trial

A phase 1 trial of YOLT-101 gene therapy demonstrated sustained reductions in PCSK9 and LDL cholesterol levels in patients with heterozygous familial hypercholesterolemia, though larger studies are required to establish clinical efficacy.

By Dr. Keala Nakamura January 5, 2026

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In Vivo PCSK9 Base Editing Demonstrates Proof-of-Concept for Familial Hypercholesterolemia: A Critical Assessment of Phase 1 Trial Results

A phase 1 clinical trial of liver-targeted PCSK9 base editing demonstrates preliminary efficacy for familial hypercholesterolemia treatment, though significant optimization challenges remain for clinical translation. The study represents an important milestone in applying CRISPR-based therapeutics to cardiovascular disease management.

By Dr. Robert Kim January 5, 2026