Does base editing of the PCSK9 gene reduce LDL cholesterol in humans?
What the Evidence Shows
We analyzed the available evidence on base editing of the PCSK9 gene and its effect on LDL cholesterol in humans. What we’ve found so far is that one assertion supports the idea that using base editing to modify the PCSK9 gene leads to lower levels of LDL cholesterol in the bloodstream [1]. No...
Evidence from Studies
Supporting (0)
No supporting evidence found
Contradicting (0)
No contradicting evidence found
Primary Studies (2)
In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial.
DOI: 10.1038/s41591-026-04254-4
Abstract 4139206: Design of Heart-2: a phase 1b clinical trial of VERVE-102, an in vivo base editing medicine delivered by a GalNAc-LNP and targeting PCSK9 to durably lower LDL cholesterol
DOI: 10.1161/circ.150.suppl_1.4139206
Update History
Published
May 26, 2026·Last updated May 26, 2026- Invalid DateNew topic created from assertion