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

Summary: 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, ena...

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Bibliographic Details
Main Authors: Jonathan D. Finn, Amy Rhoden Smith, Mihir C. Patel, Lucinda Shaw, Madeleine R. Youniss, Jane van Heteren, Tanner Dirstine, Corey Ciullo, Reynald Lescarbeau, Jessica Seitzer, Ruchi R. Shah, Aalok Shah, Dandan Ling, Jacqueline Growe, Melissa Pink, Ellen Rohde, Kristy M. Wood, William E. Salomon, William F. Harrington, Christian Dombrowski, Walter R. Strapps, Yong Chang, David V. Morrissey
Format: Article
Language:English
Published: Elsevier 2018-02-01
Series:Cell Reports
Online Access:http://www.sciencedirect.com/science/article/pii/S2211124718301827