Work

In vivo base editing of Angptl3 via lipid nanoparticles to treat cardiovascular disease

Year: 2025

Type: article

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.

Source: Molecular Therapy — Nucleic Acids

DOI: 10.1016/j.omtn.2025.102486

Authors: Jennifer Khirallah, Hanan Bloomer, Douglas Wich, Changfeng Huang, J. Noah Workman, Yamin Li, Gregory A. Newby, David R. Liu, Qiaobing Xu

Institutions: Tufts University, Johns Hopkins University, Broad Institute, Howard Hughes Medical Institute, Harvard University, Massachusetts Institute of Technology

Language: English

Topic: RNA Interference and Gene Delivery

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,900

Funders: National Science Foundation, Howard Hughes Medical Institute, National Institutes of Health, National Science Foundation Graduate Research Fellowship Program

Awards: Medical Scientist Training Program at Tufts University, G5440924496, G7832150550, G9000022816, G9000174863, G9000325443

OpenAlex ID: W4407597095

FWCI: 5.556

Cites: 49

Cited by: 22

Related to: 10