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Genetic research

Gene Therapy Shows Promise in Treating Cardiovascular Disease

Strategic Insights

— 1 min read

Scientists at Tufts University have made significant progress in developing a gene therapy that targets cardiovascular disease (CVD), one of the leading causes of death globally. By editing the gene responsible for producing a protein that contributes to high levels of blood lipids, researchers were able to achieve durable reductions in serum ANGPTL3, LDL-C, and TGs in mice. The therapy, which uses lipid nanoparticles to deliver CRISPR adenine base editors, has shown promising results in a high-fat high-cholesterol diet challenge, with blood lipid levels remaining low for up to 191 days after treatment.

Key Takeaways:

  • Researchers at Tufts University used gene therapy to target ANGPTL3, a protein that exacerbates CVD, by editing the gene responsible for its production.
  • The therapy, which involves using lipid nanoparticles to deliver CRISPR adenine base editors, achieved over 60% base editing in the liver and durable reductions in serum ANGPTL3, LDL-C, and TGs for at least 100 days.
  • The therapy was successful in reducing blood lipid levels in mice, even when challenged with a high-fat high-cholesterol diet up to 191 days after treatment.
  • The research provides a foundation for a potential one-and-done treatment for CVD.
  • The study was supported by the National Science Foundation, Howard Hughes Medical Institute, National Institutes of Health, and the National Science Foundation Graduate Research Fellowship Program.
  • Additional authors on the study include Hanan Bloomer, Douglas Wich, Changfeng Huang, J. Noah Workman, Yamin Li, Gregory A. Newby, David R. Liu, and Qiaobing Xu.

Statistics:

  • Over 60% base editing achieved in the liver
  • Durable reductions in serum ANGPTL3, LDL-C, and TGs for at least 100 days
  • Blood lipid levels remained low in mice challenged with a high-fat high-cholesterol diet up to 191 days after therapy
  • 100 days: duration of reductions in serum ANGPTL3, LDL-C, and TGs
  • 191 days: duration of challenge with high-fat high-cholesterol diet after therapy

Sources:

  • NewsRx. Tufts University Researchers Discuss Research in Gene Therapy (In vivo base editing of Angptl3 via lipid nanoparticles to treat cardiovascular disease). Cardiovascular Week. June 9, 2025; p 202.
  • In vivo base editing of Angptl3 via lipid nanoparticles to treat cardiovascular disease. Molecular Therapy: Nucleic Acids, 2025, 36(2):102486. (Molecular Therapy: Nucleic Acids - http://www.cell.com/molecular-therapy-family/nucleic-acids/latest-content).

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