Article

High-content screening for rare respiratory diseases: readthrough therapy in primary ciliary dyskinesia

2020-02-28

Abstract excerpt

Development of therapeutic approaches for rare respiratory diseases is hampered by the lack of systems that allow medium-to-high-throughput screening of fully differentiated respiratory epithelium from affected patients. This is a particular problem for primary ciliary dyskinesia (PCD), a rare genetic disease caused by mutations in genes that adversely affect ciliary movement and consequently mucociliary transport...

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Literature Corpus work: 77626eb7-9d9e-544a-9a70-c69e3b1b74d6
DOI: 10.1101/2020.02.28.959189

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