Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates

Molecular Therapy

Volumn 26, Issue 3, 2018, Pages 708-717

  Foster, Donald J ^a   Brown, Christopher R ^a   Shaikh, Sarfraz ^a   Trapp, Casey ^a   Schlegel, Mark K ^a   Qian, Kun ^a   Sehgal, Alfica ^a   Rajeev, Kallanthottathil G ^a   Jadhav, Vasant ^a   Manoharan, Muthiah ^a   Kuchimanchi, Satya ^a   Maier, Martin A ^a   Milstein, Stuart ^a  

^a ALNYLAM PHARMACEUTICALS   (United States)

Author keywords

GalNAc conjugates; RNAi; siRNA

Indexed keywords

MESSENGER RNA; N ACETYLGALACTOSAMINE; SMALL INTERFERING RNA; ARGONAUTE PROTEIN; EIF2C2 PROTEIN, MOUSE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DRUG CONJUGATION; DRUG DESIGN; DRUG EXPOSURE; DRUG POTENCY; FEMALE; GENE SILENCING; IN VITRO STUDY; IN VIVO STUDY; LIVER CELL; LIVER TISSUE; MACACA FASCICULARIS; MALE; MOUSE; NONHUMAN; POLYMERASE CHAIN REACTION; RNA INTERFERENCE; RNA SYNTHESIS; ANIMAL; CHEMISTRY; GENE EXPRESSION REGULATION; GENETICS; LIVER; METABOLISM; TRANSGENIC MOUSE;

ACETYLGALACTOSAMINE; ANIMALS; ARGONAUTE PROTEINS; GENE EXPRESSION REGULATION; GENE SILENCING; HEPATOCYTES; LIVER; MALE; MICE; MICE, TRANSGENIC; RNA INTERFERENCE; RNA, MESSENGER; RNA, SMALL INTERFERING;

EID: 85042001178     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2017.12.021     Document Type: Article

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