CFTR mRNA-Based Gene Therapy for Cystic Fibrosis: A Mutation-Agnostic Strategy to Restore Ion Transport Function
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Cystic fibrosis is a severe autosomal recessive disorder caused by mutations in the CFTR gene, leading to dysfunctional chloride ion transport across epithelial cells. This results in the accumulation of thick, sticky mucus, primarily affecting the lungs, panc... ...
