A comparative evaluation of NB30, NB54 and PTC124 in translational read-through efficacy for treatment of an USH1C nonsense mutation

EMBO Molecular Medicine

Volumn 4, Issue 11, 2012, Pages 1186-1199

  Goldmann, Tobias ^a,c   Overlack, Nora ^a   Möller, Fabian ^a   Belakhov, Valery ^b   van Wyk, Michiel ^a,d   Baasov, Timor ^b   Wolfrum, Uwe ^a   Nagel Wolfrum, Kerstin ^a  

^a JOHANNES GUTENBERG UNIVERSITY   (Germany)

^b TECHNION ISRAEL INSTITUTE OF TECHNOLOGY   (Israel)

^c UNIVERSITY OF FREIBURG   (Germany)

^d VETSUISSE FACULTY UNIVERSITY OF ZURICH   (Switzerland)

Author keywords

Drug therapy; Pharmacogenetics; Retinitis pigmentosa; Sensoneuronal degeneration; Usher syndrome

Indexed keywords

AMINOGLYCOSIDE ANTIBIOTIC AGENT; ATALUREN; HARMONIN A1; NB 30; NB 54; SCAFFOLD PROTEIN; UNCLASSIFIED DRUG;

ANIMAL TISSUE; ARTICLE; BIOCOMPATIBILITY; COMPARATIVE STUDY; CONTROLLED STUDY; DRUG EFFICACY; DRUG POTENTIATION; DRUG SAFETY; EYE TOXICITY; FLUORESCENCE MICROSCOPY; GENE; GENE FUNCTION; HUMAN; HUMAN CELL; HUMAN TISSUE; IMMUNOFLUORESCENCE TEST; MOUSE; NONHUMAN; NONSENSE MUTATION; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN FUNCTION; RETINA DISEASE; RNA TRANSLATION; TISSUE CULTURE; TRANSLATIONAL READ THROUGH; USHER SYNDROME TYPE 1C GENE; WESTERN BLOTTING;

ADAPTOR PROTEINS, SIGNAL TRANSDUCING; AMINOGLYCOSIDES; ANIMALS; CELL LINE; CODON, NONSENSE; FEMALE; HUMANS; MALE; MICE; MICE, INBRED C57BL; OXADIAZOLES; PEPTIDE CHAIN ELONGATION, TRANSLATIONAL; RETINA; RETINAL DISEASES;

EID: 84868333419     PISSN: 17574676     EISSN: 17574684     Source Type: Journal    
DOI: 10.1002/emmm.201201438     Document Type: Article

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