Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides

Amyloid

Volumn 23, Issue 3, 2016, Pages 148-157

  Ackermann, Elizabeth J ^a   Guo, Shuling ^a   Benson, Merrill D ^b   Booten, Sheri ^a   Freier, Sue ^a   Hughes, Steven G ^a   Kim, Tae Won ^a   Jesse Kwoh, T ^a   Matson, John ^a   Norris, Dan ^a   Yu, Rosie ^a   Watt, Andy ^a   Monia, Brett P ^a  

^a ISIS PHARMACEUTICALS   (United States)

^b INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Amyloidosis; ATTR; FAC; familial; FAP

Indexed keywords

C REACTIVE PROTEIN; COMPLEMENT; COMPLEMENT COMPONENT C5A; CREATINE KINASE; INOTERSEN; MESSENGER RNA; PLACEBO; PREALBUMIN; RETINOL; ANTISENSE OLIGONUCLEOTIDE; RIBONUCLEASE H; RIBONUCLEASE HI;

ADULT; AMYLOIDOSIS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTISENSE THERAPY; AREA UNDER THE CURVE; ARTICLE; BLOOD BRAIN BARRIER; BLOOD CHEMISTRY; BLOOD CLOTTING PARAMETERS; BLOOD RETINA BARRIER; CONTROLLED STUDY; CREATINE KINASE BLOOD LEVEL; DOSE RESPONSE; DOUBLE BLIND PROCEDURE; DRUG BLOOD LEVEL; DRUG DESIGN; DRUG DISPOSITION; DRUG DOSE COMPARISON; DRUG DOSE ESCALATION; DRUG METABOLISM; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; ELECTROCARDIOGRAM; ELIMINATION HALF-LIFE; FEMALE; HEADACHE; HEMATOLOGY; HEPG2 CELL LINE; HUMAN; HUMAN CELL; HUMAN TISSUE; IC50; IN VIVO STUDY; INJECTION SITE REACTION; MACACA FASCICULARIS; MALE; MAXIMUM PLASMA CONCENTRATION; MIDDLE AGED; MOUSE; MULTIPLE DRUG DOSE; MYALGIA; NEUROPROTECTION; NONHUMAN; NORMAL HUMAN; PHARMACODYNAMICS; PHASE 1 CLINICAL TRIAL; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; PROTEIN SYNTHESIS; RANDOMIZED CONTROLLED TRIAL; RNA TRANSCRIPTION; SIDE EFFECT; SINGLE DRUG DOSE; SOMNOLENCE; T WAVE INVERSION; THROMBOCYTE COUNT; TIME TO MAXIMUM PLASMA CONCENTRATION; TRANSGENIC MOUSE; TRANSTHYRETIN AMYLOIDOSIS; URINALYSIS; VITAL SIGN; VITAMIN BLOOD LEVEL; YOUNG ADULT; ADOLESCENT; AMYLOID NEUROPATHIES, FAMILIAL; ANIMAL; ANTAGONISTS AND INHIBITORS; BIOSYNTHESIS; CLINICAL TRIAL; CYTOLOGY; GENE EXPRESSION; GENETICS; HEP-G2 CELL LINE; LIVER; LIVER CELL; METABOLISM; PRIMARY CELL CULTURE; RNA CLEAVAGE;

ADOLESCENT; ADULT; AMYLOID NEUROPATHIES, FAMILIAL; ANIMALS; DOUBLE-BLIND METHOD; FEMALE; GENE EXPRESSION; HEALTHY VOLUNTEERS; HEP G2 CELLS; HEPATOCYTES; HUMANS; LIVER; MACACA FASCICULARIS; MALE; MICE; MICE, TRANSGENIC; MIDDLE AGED; OLIGONUCLEOTIDES, ANTISENSE; PREALBUMIN; PRIMARY CELL CULTURE; RIBONUCLEASE H; RNA CLEAVAGE; RNA, MESSENGER;

EID: 84976444883     PISSN: 13506129     EISSN: 17442818     Source Type: Journal    
DOI: 10.1080/13506129.2016.1191458     Document Type: Article

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