Emerging Personalized Opportunities for Enhancing Translational Readthrough in Rare Genetic Diseases and Beyond
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DOI: 10.3390/ijms24076101
Abstract: 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… read more here.
Keywords: genetic diseases; stop codon; readthrough; rare genetic ... See more keywords