In vivo base editing of Angptl3 via lipid nanoparticles to treat cardiovascular disease
Jennifer Khirallah, Hanan Bloomer, Douglas Wich, Changfeng Huang, J. Noah Workman, Yamin Li, Gregory A. Newby, David R. Liu, Qiaobing Xu
Abstract
, which we administered to mice intravenously. We achieved over 60% base editing in the liver and durable reductions in serum ANGPTL3, LDL-C, and TGs for at least 100 days. Notably, blood lipid levels remained low when mice were challenged with a high-fat high-cholesterol diet up to 191 days after therapy. These results provide a foundation for a potential one-and-done treatment for CVD.
Topics & Concepts
In vivoMedicineAtherosclerotic cardiovascular diseaseDiseaseChemistryInternal medicineBiologyBiotechnologyLipid metabolism and disordersSystemic Lupus Erythematosus ResearchPharmacological Effects of Natural Compounds