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Properties of Non-Aminoglycoside Compounds Used to Stimulate Translational Readthrough of PTC Mutations in Primary Ciliary Dyskinesia
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 readthrough of premature termi...
Autores principales: | Dabrowski, Maciej, Bukowy-Bieryllo, Zuzanna, Jackson, Claire L., Zietkiewicz, Ewa |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8125088/ https://www.ncbi.nlm.nih.gov/pubmed/34066907 http://dx.doi.org/10.3390/ijms22094923 |
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