A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing - Finn, Jonathan D.; Smith, Amy Rhoden; Patel, Mihir C.; Shaw, Lucinda; Youniss, Madeleine R.; van Heteren, Jane; Dirstine, Tanner; Ciullo, Corey; Lescarbeau, Reynald; Seitzer, Jessica; Shah, Ruchi R.; Shah, Aalok; et al. (Cell reports (Cambridge), 2018)
Keywords: Animals; Base Sequence; Cas9; CRISPR; CRISPR-Associated Protein 9 - metabolism; CRISPR-Cas Systems - genetics; CRISPR/Cas9; Gene Editing; gene therapy; Gene Transfer Techniques; genome editing; lipid nanoparticle; Lipids - chemistry; Liver - metabolism; liver delivery; LNP; Mice; Nanoparticles - administration & dosage; Nanoparticles - chemistry; Rats; RNA, Guide, CRISPR-Cas Systems - chemistry; RNA, Guide, CRISPR-Cas Systems - genetics; sgRNA; TTR; CRISPR; genome editing; lipid nanoparticle; TTR; CRISPR/Cas9; sgRNA; gene therapy; Cas9; liver delivery; LNP