Ataluren as an agent for therapeutic nonsense suppression

Annual Review of Medicine

Volumn 64, Issue , 2013, Pages 407-425

  Peltz, Stuart W ^a   Morsy, Manal ^a   Welch, Ellen M ^a   Jacobson, Allan ^b  

^a PTC THERAPEUTICS   (United States)

^b UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

Author keywords

inherited disorders; nonsense codon readthrough; nonsense codons; premature translation termination; PTC124 ataluren

Indexed keywords

AMINOGLYCOSIDE; ATALUREN; CARNITINE PALMITOYLTRANSFERASE 1A; CARNITINE PALMITOYLTRANSFERASE I; MESSENGER RNA; UNCLASSIFIED DRUG;

CYSTIC FIBROSIS; DOSE RESPONSE; DRUG MECHANISM; DUCHENNE MUSCULAR DYSTROPHY; EXON; GENE REPRESSION; GENETIC DISORDER; HUMAN; HURLER SYNDROME; MIYOSHI MYOPATHY; NEURONAL CEROID LIPOFUSCINOSIS; NONHUMAN; NONSENSE MEDIATED MRNA DECAY; NONSENSE MUTATION; PRIORITY JOURNAL; REVIEW; SIGNAL TRANSDUCTION; STOP CODON; TRANSCRIPTION TERMINATION; USHER SYNDROME;

ANIMALS; CODON, NONSENSE; DISEASE; HUMANS; NONSENSE MEDIATED MRNA DECAY; OXADIAZOLES; RNA STABILITY; TRANSCRIPTION, GENETIC;

EID: 84873020912     PISSN: 00664219     EISSN: 1545326X     Source Type: Book Series    
DOI: 10.1146/annurev-med-120611-144851     Document Type: Review

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