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

Translational read-through-inducing drugs (TRIDs) promote read-through of nonsense mutations, placing them in the spotlight of current gene-based therapeutic research. Here, we compare for the first time the relative efficacies of new-generation aminoglycosides NB30, NB54 and the chemical compound P...

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Главные авторы: Goldmann, Tobias, Overlack, Nora, Möller, Fabian, Belakhov, Valery, van Wyk, Michiel, Baasov, Timor, Wolfrum, Uwe, Nagel-Wolfrum, Kerstin
Формат: Artigo
Язык:Inglês
Опубликовано: WILEY-VCH Verlag 2012
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Research Articles
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC3494875/
https://ncbi.nlm.nih.gov/pubmed/23027640
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/emmm.201201438
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