Evaluation of Aminoglycoside and Non-Aminoglycoside Compounds for Stop-Codon Readthrough Therapy in Four Lysosomal Storage Diseases

Nonsense mutations are quite prevalent in inherited diseases. Readthrough drugs could provide a therapeutic option for any disease caused by this type of mutation. Geneticin (G418) and gentamicin were among the first to be described. Novel compounds have been generated, but only a few have shown imp...

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Detalles Bibliográficos
Autores principales: Gómez-Grau, Marta, Garrido, Elena, Cozar, Mónica, Rodriguez-Sureda, Víctor, Domínguez, Carmen, Arenas, Concepción, Gatti, Richard A., Cormand, Bru, Grinberg, Daniel, Vilageliu, Lluïsa
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2015
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4545610/
https://www.ncbi.nlm.nih.gov/pubmed/26287674
http://dx.doi.org/10.1371/journal.pone.0135873

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4545610/
https://www.ncbi.nlm.nih.gov/pubmed/26287674
http://dx.doi.org/10.1371/journal.pone.0135873

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