A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

• Published in: Cell reports (Cambridge) Vol. 22; no. 9; pp. 2227 - 2235
• Main Authors: 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, Ling, Dandan, Growe, Jacqueline, Pink, Melissa, Rohde, Ellen, Wood, Kristy M., Salomon, William E., Harrington, William F., Dombrowski, Christian, Strapps, Walter R., Chang, Yong, Morrissey, David V.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 27.02.2018; Elsevier
• Subjects: 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

The development of clinically viable delivery methods presents one of the greatest challenges in the therapeutic application of CRISPR/Cas9 mediated genome editing. Here, we report the development of a lipid nanoparticle (LNP)-mediated delivery system that, with a single administration, enabled sign...