Lipid nanoparticles incorporating a GalNAc ligand enable in vivo liver ANGPTL3 editing in wild-type and somatic LDLR knockout non-human primates
Lisa N. Kasiewicz, Souvik Biswas, Aaron Beach, Huilan Ren, Chaitali Dutta, Anne Marie Mazzola, Ellen Rohde, Alexandra C. Chadwick, Christopher Cheng, Kiran Musunuru, Sekar Kathiresan, Padma Malyala, Kallanthottathil G. Rajeev, Andrew M. Bellinger
This study develops GalNAc-LNPs for liver gene editing in NHPs, achieving high editing efficiency and ANGPTL3 reduction in both HoFH and wild-type models.
bioRxiv · Nov 8, 2021 · PreprintGenetics