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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...
Autores principales: | Wagner, Roland N., Wießner, Michael, Friedrich, Andreas, Zandanell, Johanna, Breitenbach-Koller, Hannelore, Bauer, Johann W. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2023
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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 |
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