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Article

Precise Targeting of miRNA Sites Restores CFTR Activity in CF Bronchial Epithelial Cells

5 Feb 2020

Abstract excerpt

MicroRNAs that are overexpressed in cystic fibrosis (CF) bronchial epithelial cells (BEC) negatively regulate CFTR and nullify the beneficial effects of CFTR modulators. We hypothesized that it is possible to reverse microRNA-mediated inhibition of CFTR using CFTR-specific target site blockers (TSBs) and to develop a drug-device combination inhalation therapy for CF. Lead microRNA expression was quantified in a...

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PMID: 32059764
DOI: 10.1016/j.ymthe.2020.02.001

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