SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice

Nature Chemical Biology

Volumn 11, Issue 7, 2015, Pages 511-517

  Palacino, James ^a   Swalley, Susanne E ^a   Song, Cheng ^a   Cheung, Atwood K ^a   Shu, Lei ^a   Zhang, Xiaolu ^a   Van Hoosear, Mailin ^a   Shin, Youngah ^a   Chin, Donovan N ^b   Keller, Caroline Gubser ^b   Beibel, Martin ^a   Renaud, Nicole A ^a   Smith, Thomas M ^a   Salcius, Michael ^a   Shi, Xiaoying ^a   Hild, Marc ^a   Servais, Rebecca ^a   Jain, Monish ^a   Deng, Lin ^a   Bullock, Caroline ^a   McLellan, Michael ^a   Schuierer, Sven ^b   Murphy, Leo ^a   Blommers, Marcel J J ^b   Blaustein, Cecile ^a   Berenshteyn, Frada ^a   Lacoste, Arnaud ^a   Thomas, Jason R ^a   Roma, Guglielmo ^b   Michaud, Gregory A ^a   Tseng, Brian S ^a   Porter, Jeffery A ^a   Myer, Vic E ^a   Tallarico, John A ^a   Hamann, Lawrence G ^a   Curtis, Daniel ^a   Fishman, Mark C ^a   Dietrich, William F ^a   Dales, Natalie A ^a   Sivasankaran, Rajeev ^a   more..

^a NOVARTIS INSTITUTES FOR BIOMEDICAL RESEARCH   (United States)

^b NOVARTIS PHARMA AG   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

2 [6 [METHYL (2,2,6,6 TETRAMETHYLPIPERIDIN 4 YL)AMINO]PYRIDAZIN 3 YL] 5 (1H PYRAZOL 4 YL)PHENOL; 3 FLUORO 4 [6 [METHYL (2,2,6,6 TETRAMETHYLPIPERIDIN 4 YL)AMINO]PYRIDAZIN 3 YL]PHENOL; 4 [(1H IMIDAZOL 1 YL)METHYL] 2 [6 [METHYL (2,2,6,6 TETRAMETHYLPIPERIDIN 4 YL)AMINO]PYRIDAZIN 3 YL]PHENOL; 5 (1H PYRAZOL 4 YL) 2 [6 [(2,2,6,6 TETRAMETHYLPIPERIDIN 4 YL)OXY]PYRIDAZIN 3 YL]PHENOL; DOUBLE STRANDED RNA; MESSENGER RNA PRECURSOR; PYRIDAZINE DERIVATIVE; SMALL MOLECULE TRANSPORT AGENT; SMALL NUCLEAR RIBONUCLEOPROTEIN; SURVIVAL MOTOR NEURON PROTEIN 2; UNCLASSIFIED DRUG; PROTEIN BINDING; RIBONUCLEOPROTEIN, U1 SMALL NUCLEAR; RNA PRECURSORS; RNA, DOUBLE-STRANDED; SMALL MOLECULE LIBRARIES; SMN2 PROTEIN, MOUSE; SNRNP70 PROTEIN, MOUSE; SURVIVAL OF MOTOR NEURON 2 PROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BINDING AFFINITY; CONTROLLED STUDY; DRUG BINDING; DRUG BIOAVAILABILITY; DRUG BLOOD LEVEL; DRUG DISTRIBUTION; DRUG EFFICACY; DRUG HALF LIFE; DRUG MECHANISM; DRUG SYNTHESIS; HUMAN; HUMAN CELL; IN VIVO STUDY; MOLECULAR RECOGNITION; MOUSE; NONHUMAN; PRIORITY JOURNAL; RNA STRUCTURE; SINGLE DRUG DOSE; SPINAL MUSCULAR ATROPHY; AGONISTS; ALTERNATIVE SPLICING; ANIMALS; BINDING SITES; CHEMICAL SYNTHESIS; CHEMISTRY; DISEASE MODELS, ANIMAL; DRUG EFFECTS; FEMALE; GENE EXPRESSION; GENETICS; HUMANS; METABOLISM; MICE; MICE, TRANSGENIC; MODELS, MOLECULAR; MORTALITY; MUSCULAR ATROPHY, SPINAL; PATHOLOGY; PHARMACOLOGY; PROTEIN STABILITY; PROTEOLYSIS; SMALL MOLECULE LIBRARIES; SURVIVAL ANALYSIS;

MUS;

ALTERNATIVE SPLICING; ANIMALS; BINDING SITES; DISEASE MODELS, ANIMAL; FEMALE; GENE EXPRESSION; HUMANS; MICE; MICE, TRANSGENIC; MODELS, MOLECULAR; MUSCULAR ATROPHY, SPINAL; PROTEIN BINDING; PROTEIN STABILITY; PROTEOLYSIS; RIBONUCLEOPROTEIN, U1 SMALL NUCLEAR; RNA PRECURSORS; RNA, DOUBLE-STRANDED; SMALL MOLECULE LIBRARIES; SURVIVAL ANALYSIS; SURVIVAL OF MOTOR NEURON 2 PROTEIN;

EID: 84931565562     PISSN: 15524450     EISSN: 15524469     Source Type: Journal    
DOI: 10.1038/nchembio.1837     Document Type: Article

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