Hepatocyte-Specific Delivery of siRNAs Conjugated to Novel Non-nucleosidic Trivalent N-Acetylgalactosamine Elicits Robust Gene Silencing in Vivo

ChemBioChem

Volumn 16, Issue 6, 2015, Pages 903-908

  Rajeev, Kallanthottathil G ^a   Nair, Jayaprakash K ^a   Jayaraman, Muthusamy ^a   Charisse, Klaus ^a   Taneja, Nate ^a   O'Shea, Jonathan ^a   Willoughby, Jennifer L S ^a   Yucius, Kristina ^a   Nguyen, Tuyen ^a   Shulga Morskaya, Svetlana ^a   Milstein, Stuart ^a   Liebow, Abigail ^a   Querbes, William ^a   Borodovsky, Anna ^a   Fitzgerald, Kevin ^a   Maier, Martin A ^a   Manoharan, Muthiah ^a  

^a ALNYLAM PHARMACEUTICALS   (United States)

Author keywords

asialoglycoprotein receptor (ASGPR); N acetylgalactosamine (GalNAc); oligonucleotide conjugates; RNAi; siRNA

Indexed keywords

CARBOXYLIC ACID; DICHLOROMETHANE; GALACTOSAMINE; LIPOFECTAMINE; MONOMER; N ACETYLGALACTOSAMINE; OLIGONUCLEOTIDE; PHOSPHATE BUFFERED SALINE; PHOSPHORAMIDOUS ACID; SMALL INTERFERING RNA; SUCCINIC ANHYDRIDE; TRIETHYLAMINE; DRUG CARRIER; PREALBUMIN;

ANIMAL EXPERIMENT; ARTICLE; BINDING AFFINITY; CONTROLLED STUDY; DRUG CONJUGATION; DRUG DELIVERY SYSTEM; DRUG SYNTHESIS; ENZYME LINKED IMMUNOSORBENT ASSAY; GENE SILENCING; HIGH PERFORMANCE LIQUID CHROMATOGRAPHY; IN VITRO STUDY; IN VIVO STUDY; LIVER CELL; MOLECULAR WEIGHT; MOUSE; NONHUMAN; PRIORITY JOURNAL; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SOLID PHASE SYNTHESIS; SPATIAL ORIENTATION; ANIMAL; CHEMISTRY; DEFICIENCY; GENETICS; METABOLISM;

ACETYLGALACTOSAMINE; ANIMALS; DRUG CARRIERS; GENE SILENCING; HEPATOCYTES; MICE; PREALBUMIN; RNA, SMALL INTERFERING;

EID: 84926320054     PISSN: 14394227     EISSN: 14397633     Source Type: Journal    
DOI: 10.1002/cbic.201500023     Document Type: Article

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