CRISPR gene editing of angiopoietin-like 3: toward one-time precision therapy for dyslipidaemia
Abstract
In a landmark study published in The New England Journal of Medicine , Laffin et al. 1 present the first-in-human clinical evaluation of CTX310, an investigational in vivo CRISPR–Cas9 therapy engineered to induce permanent loss-of-function mutations in the hepatic ANGPTL3 gene, resulting in sustained lowering of atherogenic lipoproteins. This marks a major step toward durable, potentially lifelong lipid lowering through a single therapeutic intervention, 1 potentially replacing years of ongoing lipid-lowering treatment for atherosclerotic cardiovascular disease (ASCVD) patients.
Fields of Study & Subjects
Lipid metabolism and disorders
Angiogenesis and VEGF in Cancer
Systemic Lupus Erythematosus Research
Publication details
- Journal / source
- Signal Transduction and Targeted Therapy
- Publisher
- Springer Science and Business Media LLC
- Volume
- 11
- Issue
- 1
- Publication date
- 4 March 2026
- Language
- EN