Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression

Molecular Therapy

Volumn 26, Issue 1, 2018, Pages 105-114

  Willoughby, Jennifer L S ^a   Chan, Amy ^a   Sehgal, Alfica ^a   Butler, James S ^a   Nair, Jayaprakash K ^a   Racie, Tim ^a   Shulga Morskaya, Svetlana ^a   Nguyen, Tuyen ^a   Qian, Kun ^a   Yucius, Kristina ^a   Charisse, Klaus ^a   van Berkel, Theo J C ^b   Manoharan, Muthiah ^a   Rajeev, Kallanthottathil G ^a   Maier, Martin A ^a   Jadhav, Vasant ^a   Zimmermann, Tracy S ^a  

^a ALNYLAM PHARMACEUTICALS   (United States)

^b LEIDEN UNIVERSITY   (Netherlands)

Author keywords

asialoglycoprotein receptor; GalNAc siRNA; RNA interference

Indexed keywords

ASIALOGLYCOPROTEIN RECEPTOR; N ACETYLGALACTOSAMINE; SMALL INTERFERING RNA; DRUG CARRIER;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DRUG BLOOD LEVEL; DRUG DELIVERY SYSTEM; DRUG LIVER LEVEL; DRUG RECEPTOR BINDING; FEMALE; HUMAN; HUMAN TISSUE; INFANT; LIVER DISEASE; MAXIMUM PLASMA CONCENTRATION; MOUSE; NONHUMAN; PHARMACODYNAMICS; PROTEIN EXPRESSION; SINGLE DRUG DOSE; ANIMAL; CHEMISTRY; DISEASE MODEL; GENE EXPRESSION REGULATION; GENE SILENCING; GENETICS; KNOCKOUT MOUSE; LIVER CELL; LIVER CIRRHOSIS; METABOLISM; PATHOLOGY; PRECLINICAL STUDY; PROTEIN SUBUNIT; RNA INTERFERENCE;

ACETYLGALACTOSAMINE; ANIMALS; ASIALOGLYCOPROTEIN RECEPTOR; DISEASE MODELS, ANIMAL; DRUG CARRIERS; DRUG DELIVERY SYSTEMS; DRUG EVALUATION, PRECLINICAL; FEMALE; GENE EXPRESSION REGULATION; GENE SILENCING; HEPATOCYTES; HUMANS; LIVER CIRRHOSIS; MICE; MICE, KNOCKOUT; PROTEIN SUBUNITS; RNA INTERFERENCE; RNA, SMALL INTERFERING;

EID: 85030565780     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2017.08.019     Document Type: Article

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