Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine to Treat Spinal Muscular Atrophy

Journal of Medicinal Chemistry

Volumn 59, Issue 13, 2016, Pages 6086-6100

  Ratni, Hasane ^a   Karp, Gary M ^b   Weetall, Marla ^b   Naryshkin, Nikolai A ^b   Paushkin, Sergey V ^c   Chen, Karen S ^c   McCarthy, Kathleen D ^c   Qi, Hongyan ^b   Turpoff, Anthony ^b   Woll, Matthew G ^b   Zhang, Xiaoyan ^b   Zhang, Nanjing ^b   Yang, Tianle ^b   Dakka, Amal ^b   Vazirani, Priya ^b   Zhao, Xin ^b   Pinard, Emmanuel ^a   Green, Luke ^a   David Pierson, Pascale ^a   Tuerck, Dietrich ^a   Poirier, Agnes ^a   Muster, Wolfgang ^a   Kirchner, Stephan ^a   Mueller, Lutz ^a   Gerlach, Irene ^a   Metzger, Friedrich ^a   more..

^a F HOFFMANN LA ROCHE LTD   (Switzerland)

^b PTC THERAPEUTICS   (United States)

^c SMA Foundation   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

2 (1 METHYLBENZIMIDAZOL 5 YL) 7 (4 METHYLPIPERAZIN 1 YL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 (2 METHYL 1,3 BENZOXAZOL 6 YL) 7 (4 METHYLPIPERAZIN 1 YL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 (2 METHYLIMIDAZO[1,2 A]PYRIDIN 6 YL) 7 (4 METHYLPIPERAZIN 1 YL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 (2,8 DIMETHYLIMIDAZO[1,2 A]PYRIDIN 6 YL) 7 (4 METHYLPIPERAZIN 1 YL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 (4 ETHYL 6 METHYLPYRAZOLO[1,5 A]PYRAZIN 2 YL) 9 METHYL 7 (1 METHYL 4 PIPERIDYL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 (4 ETHYL 6 METHYLPYRAZOLO[1,5 A]PYRAZIN 2 YL) 9 METHYL 7 (1,2,3,6 TETRAHYDROPYRIDIN 4 YL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 (4 FLUORO 2 METHYL 1,3 BENZOXAZOL 6 YL) 7 (4 METHYLPIPERAZIN 1 YL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 (4 FLUORO 2 METHYL 1,3 BENZOXAZOL 6 YL) 7 PIPERAZIN 1 YLPYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 (4,6 DIMETHYLPYRAZOLO[1,5 A]PYRAZIN 2 YL) 7 (1 ETHYL 4 PIPERIDYL) 9 METHYLPYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 (4,6 DIMETHYLPYRAZOLO[1,5 A]PYRAZIN 2 YL) 7 (4 METHYLPIPERAZIN 1 YL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 (4,6 DIMETHYLPYRAZOLO[1,5 A]PYRAZIN 2 YL) 9 METHYL 7 (1 METHYL 4 PIPERIDYL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 (4,6 DIMETHYLPYRAZOLO[1,5 A]PYRAZIN 2 YL) 9 METHYL 7 (4 PIPERIDYL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 (8 FLUORO 2 METHYLIMIDAZO[1,2 A]PYRIDIN 6 YL) 7 (4 METHYLPIPERAZIN 1 YL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 (8 FLUORO 2 METHYLIMIDAZO[1,2 A]PYRIDIN 6 YL) 7 PIPERAZIN 1 YLPYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 IMIDAZO[1,2 A]PYRIDIN 6 YL 7 (4 METHYLPIPERAZIN 1 YL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 2 [2 METHYL 8 (TRIFLUOROMETHYL)IMIDAZO[1,2 A]PYRIDIN 6 YL] 7 (4 PYRROLIDIN 1 YL 1 PIPERIDYL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 7 (3,4,6,7,8,8A HEXAHYDRO 1H PYRROLO[1,2 A]PYRAZIN 2 YL) 2 (4 ETHYL 6 METHYLPYRAZOLO[1,5 A]PYRAZIN 2 YL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 7 (4 METHYLPIPERAZIN 1 YL) 2 (2 METHYL[1,2,4]TRIAZOLO[1,5 A]PYRIDIN 6 YL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; 7 [4 (AZETIDIN 1 YL) 1 PIPERIDYL] 2 (1,3 DIMETHYLPYRROLO[1,2 A]PYRAZIN 7 YL)PYRIDO[1,2 A]PYRIMIDIN 4 ONE; AGENTS AFFECTING PROTEIN METABOLISM; BRAIN PROTEIN; CENTRAL NERVOUS SYSTEM AGENTS; MESSENGER RNA; PYRIDONE DERIVATIVE; PYRIMIDINONE DERIVATIVE; SURVIVAL MOTOR NEURON PROTEIN 2; UNCLASSIFIED DRUG;

ADULT; ALTERNATIVE RNA SPLICING; AMES TEST; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BACTERIAL GROWTH; CONTROLLED STUDY; DOSE RESPONSE; DRUG BIOAVAILABILITY; DRUG CLEARANCE; DRUG DISPOSITION; DRUG EFFICACY; DRUG MEGADOSE; DRUG METABOLISM; DRUG SYNTHESIS; GENOTOXICITY; HALF LIFE TIME; HUMAN; IN VITRO STUDY; LIVER CELL; LIVER MICROSOME; LOW DRUG DOSE; MACACA FASCICULARIS; MOTONEURON; MOUSE; MUTAGENICITY; NEWBORN; NONHUMAN; PHARMACODYNAMICS; QUADRICEPS FEMORIS MUSCLE; RAT; REVERTANT; SINGLE DRUG DOSE; SPINAL MUSCULAR ATROPHY; VOLUME OF DISTRIBUTION; WEIGHT GAIN; ANIMAL; CHEMISTRY; DRUG EFFECTS; EXON; GENETICS; MUSCULAR ATROPHY, SPINAL;

ALTERNATIVE SPLICING; ANIMALS; EXONS; HUMANS; MICE; MUSCULAR ATROPHY, SPINAL; PYRIMIDINONES; RNA, MESSENGER; SURVIVAL OF MOTOR NEURON 2 PROTEIN;

EID: 84978731539     PISSN: 00222623     EISSN: 15204804     Source Type: Journal    
DOI: 10.1021/acs.jmedchem.6b00459     Document Type: Article

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