Primary ciliary dyskinesia (PCD) is a rare disease with autosomal recessive inheritance, caused mostly by bi-allelic gene mutations that impair motile cilia structure and function. Currently, there are no causal treatments for PCD. In many disease models, translational read through of premature termination codons (PTC-readthrough) induced by aminoglycosides has been proposed as an effective way of restoring functional protein expression and reducing disease symptoms. However, variable outcomes of pre-clinical trials and toxicity associated with long-term use of aminoglycosides prompt the search for other compounds that might overcome these problems. The authors tested a group of chemical compounds with known PTC-readthrough potential, collectively referred to as non-aminoglycosides (NAGs). The NAGs did not compromise the viability of the primary nasal respiratory epithelial cells. The study allowed to select of compounds with a minimal negative impact on cell viability and function but still the potential to induce PTC-readthrough.

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