Amplified genome editing by in vivo editor production

NANITE strategy quadruples genome editing efficiency in cells and triples liver editing levels in mice by enabling enzyme transfer between cells.

• Why it matters: Delivering genome editing enzymes effectively in vivo is difficult because only some cells can be transfected, limiting therapeutic impact.
• What they did: Researchers developed NANITE, a nanoparticle-based method that promotes transfer of editing enzymes between cells, tested in cultured cells and mice with a single intravenous injection.
• The result: This approach significantly enhances editing efficiency and reduces serum TTR levels, offering a promising nonviral method to amplify therapeutic genome editing effects.