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Emerging Personalized Opportunities for Enhancing Translational Readthrough in Rare Genetic Diseases and Beyond

Nonsense mutations trigger premature translation termination and often give rise to prevalent and rare genetic diseases. Consequently, the pharmacological suppression of an unscheduled stop codon represents an attractive treatment option and is of high clinical relevance. At the molecular level, the...

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Detalles Bibliográficos
Autores principales: Wagner, Roland N., Wießner, Michael, Friedrich, Andreas, Zandanell, Johanna, Breitenbach-Koller, Hannelore, Bauer, Johann W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10093890/
https://www.ncbi.nlm.nih.gov/pubmed/37047074
http://dx.doi.org/10.3390/ijms24076101