In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial

In a phase 1 trial enrolling six patients with heterozygous familial hypercholesterolemia, treatment with lipid nanoparticles designed for base editing and inactivation of PCSK9 in hepatocytes showed preliminary evidence of reduced low-density lipoprotein levels without the occurrence of serious adverse events or evidence of off-target editing.