Toward a rationale for the PTC124 (Ataluren) promoted readthrough of premature stop codons: A computational approach and GFP-reporter cell-based assay

Molecular Pharmaceutics

Volumn 11, Issue 3, 2014, Pages 653-664

  Lentini, Laura ^a   Melfi, Raffaella ^a   Di Leonardo, Aldo ^a,b   Spinello, Angelo ^a   Barone, Giampaolo ^a,c   Pace, Andrea ^a,c   Palumbo Piccionello, Antonio ^a   Pibiri, Ivana ^a  

^a UNIVERSITY OF PALERMO   (Italy)

^b Centro di OncoBiologia Sperimentale (COBS)   (Italy)

^c Istituto Euro Mediterraneo di Scienza e Tecnologia IEMEST   (Italy)

Author keywords

ataluren; cystic fibrosis (CF); Duchenne muscular distrophy (DMD); green fluorescent protein (GFP); molecular dynamics (MD); nonsense mutation readthrough; oxadiazoles; premature termination codons (PTC)

Indexed keywords

ATALUREN; COMPLEMENTARY DNA; GREEN FLUORESCENT PROTEIN; HISTONE H2B; MESSENGER RNA; CFTR PROTEIN, HUMAN; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; OXADIAZOLE DERIVATIVE; STOP CODON;

ARTICLE; CELL ASSAY; CONTROLLED STUDY; CYSTIC FIBROSIS TRANSMEMBRANE REGULATOR GENE; FEMALE; GENE CONTROL; HISTONE CODE; HUMAN; HUMAN CELL; IN VITRO STUDY; MATHEMATICAL ANALYSIS; MOLECULAR DYNAMICS; NONSENSE MUTATION; NUCLEOTIDE SEQUENCE; PLASMID; PREMATURE STOP CODON; PRIORITY JOURNAL; REGULATOR GENE; REPORTER CELL BASED ASSAY; REPORTER GENE; SITE DIRECTED MUTAGENESIS; STOP CODON; CELL CULTURE; CHEMISTRY; CONFORMATION; GENETICS; HELA CELL LINE; METABOLISM; MUTATION; PROTEIN CONFORMATION; REAL TIME POLYMERASE CHAIN REACTION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; WESTERN BLOTTING;

BLOTTING, WESTERN; CELLS, CULTURED; CODON, NONSENSE; CODON, TERMINATOR; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; GREEN FLUORESCENT PROTEINS; HELA CELLS; HUMANS; MOLECULAR DYNAMICS SIMULATION; MUTAGENESIS, SITE-DIRECTED; MUTATION; NUCLEIC ACID CONFORMATION; OXADIAZOLES; PROTEIN CONFORMATION; REAL-TIME POLYMERASE CHAIN REACTION; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA, MESSENGER;

EID: 84900364018     PISSN: 15438384     EISSN: 15438392     Source Type: Journal    
DOI: 10.1021/mp400230s     Document Type: Article

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72. This article was published after the first submission date of the present manuscript: McElroy, S. P.; Nomura, T.; Torrie, L. S.; Warbrick, E.; Gartner, U.; Wood, G.; McLean, W. H. I. A lack of premature termination codon read-through efficacy of PTC124 (Ataluren) in a diverse array of reporter assays PLoS Biol. 2013, 11, e1001593