Durability of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 with CTX310

New England Journal of Medicine 2026

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Authors: Luke J. Laffin, Stephen J. Nicholls, Russell S. Scott, Peter M. Clifton, Renate Koops, Ashish Sarraju, Shweta Singh, Qiuqing Wang, Kathy Wolski, Huansheng Xu, Jen Nielsen, Naimish Patel
+2 more Jason M. Duran, Steven E. Nissen
DOI: 10.1056/nejmc2609825
Cited by: 1
References: 4

Abstract

In a phase 1a trial, one-time CRISPR-Cas9 gene editing with CTX310 was associated with no treatment-related dose-limiting toxic effects and with sustained reductions in ANGPTL3 and atherogenic lipids at 1 year.

Fields of Study & Subjects

Lipid metabolism and disorders CRISPR and Genetic Engineering Genetic Neurodegenerative Diseases

Publication details

Journal / source
New England Journal of Medicine
Publisher
Massachusetts Medical Society
Volume
395
Issue
13
Pages
1343-1345
Publication date
28 August 2026
Language
EN