Cas9 Protein Works Better Than mRNA for Lipid Nanoparticle Delivery in Fixing CFTR

For lipid nanoparticle CFTR gene repair, delivering Cas9 as protein performed better than delivering Cas9 as mRNA.

Comparative preclinical LNP gene-editing study with in vitro and in vivo experiments found Cas9 protein LNPs outperformed Cas9 mRNA LNPs for lung editing, corrective CFTR editing, and functional CFTR recovery.

This is preclinical in vitro and in vivo work rather than human clinical evidence, so therapeutic benefit in patients with cystic fibrosis remains unproven.