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

Cell Reports

Volumn 22, Issue 9, 2018, Pages 2227-2235

  Finn, Jonathan D ^a   Smith, Amy Rhoden ^a   Patel, Mihir C ^a   Shaw, Lucinda ^a   Youniss, Madeleine R ^a   van Heteren, Jane ^a   Dirstine, Tanner ^a   Ciullo, Corey ^a   Lescarbeau, Reynald ^a   Seitzer, Jessica ^a   Shah, Ruchi R ^a   Shah, Aalok ^a   Ling, Dandan ^a   Growe, Jacqueline ^a   Pink, Melissa ^a   Rohde, Ellen ^a   Wood, Kristy M ^a   Salomon, William E ^a   Harrington, William F ^a   Dombrowski, Christian ^a   Strapps, Walter R ^a   Chang, Yong ^a   Morrissey, David V ^a   more..

^a INTELLIA THERAPEUTICS   (United States)

Author keywords

Cas9; CRISPR; CRISPR Cas9; gene therapy; genome editing; lipid nanoparticle; liver delivery; LNP; sgRNA; TTR

Indexed keywords

PREALBUMIN; RNA; SOLID LIPID NANOPARTICLE; GUIDE RNA; LIPID; NANOPARTICLE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BIODEGRADATION; CHEMICAL MODIFICATION; CONTROLLED STUDY; CRISPR CAS SYSTEM; CRISPR CAS9 SYSTEM; DRUG DELIVERY SYSTEM; GENE EDITING; IN VIVO STUDY; LIVER; LIVER CELL; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; RAT MODEL; TTR GENE; WESTERN BLOTTING; ANIMAL; CHEMISTRY; GENE TRANSFER; GENETICS; METABOLISM; NUCLEOTIDE SEQUENCE; RAT;

ANIMALS; BASE SEQUENCE; CRISPR-ASSOCIATED PROTEIN 9; CRISPR-CAS SYSTEMS; GENE EDITING; GENE TRANSFER TECHNIQUES; LIPIDS; LIVER; MICE; NANOPARTICLES; RATS; RNA, GUIDE;

EID: 85042609128     PISSN: None     EISSN: 22111247     Source Type: Journal    
DOI: 10.1016/j.celrep.2018.02.014     Document Type: Article

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