Identification and characterization of modified antisense oligonucleotides targeting DMPK in mice and nonhuman primates for the treatment of myotonic dystrophy type 1s

Journal of Pharmacology and Experimental Therapeutics

Volumn 355, Issue 2, 2015, Pages 329-340

  Pandey, Sanjay K ^a   Wheeler, Thurman M ^b,c   Justice, Samantha L ^a   Kim, Aneeza ^a   Younis, Husam S ^a   Gattis, Danielle ^a   Jauvin, Dominic ^d   Puymirat, Jack ^d   Swayze, Eric E ^a   Freier, Susan M ^a   Bennett, C Frank ^a   Thornton, Charles A ^b   MacLeod, A Robert ^a  

^a ISIS PHARMACEUTICALS   (United States)

^b UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

^c MASSACHUSETTS GENERAL HOSPITAL   (United States)

^d LAVAL UNIVERSITY   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ISIS 486178; MESSENGER RNA; MYOTONIC DYSTROPHY PROTEIN KINASE; UNCLASSIFIED DRUG; OLIGONUCLEOTIDE;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CARDIOTOXICITY; CONTROLLED STUDY; DIABETOGENESIS; DRUG DESIGN; DRUG POTENCY; DRUG TARGETING; DRUG TOLERABILITY; ENZYME INHIBITION; FEMALE; GAIN OF FUNCTION MUTATION; HAPLORHINI; HEART MUSCLE; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; INSULIN DEPENDENT DIABETES MELLITUS; LIVER; MACACA FASCICULARIS; MALE; MOUSE; MYOTONIC DYSTROPHY; NONHUMAN; PRIMATE; PRIORITY JOURNAL; SKELETAL MUSCLE; TRANSGENIC MOUSE; WILD TYPE MOUSE; ANIMAL; ANTAGONISTS AND INHIBITORS; CELL LINE; CHEMISTRY; ENZYMOLOGY; GENETICS; METABOLISM; RAT; SPRAGUE DAWLEY RAT;

ANIMALS; CELL LINE; HUMANS; MACACA FASCICULARIS; MALE; MICE; MICE, TRANSGENIC; MUSCLE, SKELETAL; MYOTONIC DYSTROPHY; MYOTONIN-PROTEIN KINASE; OLIGONUCLEOTIDES; OLIGONUCLEOTIDES, ANTISENSE; RATS; RATS, SPRAGUE-DAWLEY; RNA, MESSENGER;

EID: 84946543730     PISSN: 00223565     EISSN: 15210103     Source Type: Journal    
DOI: 10.1124/jpet.115.226969     Document Type: Article

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