Article
Inhaled delivery of a lipid nanoparticle encapsulated messenger RNA encoding a ciliary protein for the treatment of primary ciliary dyskinesia.
Pulmonary pharmacology & therapeutics - 1 Aug 2022
Woo Caroline J, Allawzi Ayed, Clark Nicholas, Kaushal Neha, Efthymiou Tim, Thamsen Maike, Nguyen Jane, Wooster Richard, Sullivan James C
Abstract excerpt
Primary ciliary dyskinesia (PCD) is a respiratory disease caused by dysfunction of the cilia with currently no approved treatments. This predominantly autosomal recessive disease is caused by mutations in any one of over 50 genes involved in cilia function; DNAI1 is one of the more frequently mutated genes, accounting for approximately 5-10% of diagnosed PCD cases. A codon-optimized mRNA encoding DNAI1 and...
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