Yang and colleagues demonstrate that lipid nanoparticle-mediated precise hepatic ASGR1 base editing safely produces robust and durable lipid lowering in an LDLR-deficient mouse model of familial hypercholesterolemia. Their work further benchmarks the lipid-lowering effects of ASGR1 and ANGPTL3 editing and supports combined ASGR1/ANGPTL3 targeting for enhanced cholesterol lowering.

Nature, Published online: 08 April 2026; doi:10.1038/s41586-026-10342-9A clinical phase 1 trial of a single infusion of CS-101, CD34+ cells modified using a transformer base editor to reactivate fetal haemoglobin production, led to early and enduring transfusion independence in patients with β-thalassaemia.