Journal
HUMAN GENE THERAPY
Volume 22, Issue 5, Pages 537-547Publisher
MARY ANN LIEBERT INC
DOI: 10.1089/hum.2010.067
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Funding
- European Community [241955]
- Deutsche Forschungsgemeinschaft (DFG)
- FAUN-Stiftung, Nurnberg
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We investigated the therapeutic potential of the premature termination codon (PTC) readthrough-inducing drug PTC124 in treating the retinal phenotype of Usher syndrome, caused by a nonsense mutation in the USH1C gene. Applications in cell culture, organotypic retina cultures, and mice in vivo revealed significant readthrough and the recovery of protein function. In comparison with other readthrough drugs, namely the clinically approved readthrough-inducing aminoglycoside gentamicin, PTC124 exhibits significant better retinal biocompatibility. Its high readthrough efficiency in combination with excellent biocompatibility makes PTC124 a promising therapeutic agent for PTCs in USH1C, as well as other ocular and nonocular genetic diseases.
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