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

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...

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Veröffentlicht in:	Molecular therapy 2020-04, Vol.28 (4), p.1190-1199
Hauptverfasser:	De Santi, Chiara, Fernández Fernández, Elena, Gaul, Rachel, Vencken, Sebastian, Glasgow, Arlene, Oglesby, Irene K., Hurley, Killian, Hawkins, Finn, Mitash, Nilay, Mu, Fangping, Raoof, Rana, Henshall, David C., Cutrona, Meritxell B., Simpson, Jeremy C., Harvey, Brian J., Linnane, Barry, McNally, Paul, Cryan, Sally Ann, MacLoughlin, Ronan, Swiatecka-Urban, Agnieszka, Greene, Catherine M.
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult ALI culture Aminophenols - pharmacology Aminopyridines - pharmacology Benzodioxoles - pharmacology Bronchi - cytology Bronchi - drug effects Bronchi - metabolism Cells, Cultured CFTR CFTR modulators Child Child, Preschool Cystic Fibrosis - genetics Cystic Fibrosis - metabolism Cystic Fibrosis - therapy Cystic Fibrosis Transmembrane Conductance Regulator - genetics Cystic Fibrosis Transmembrane Conductance Regulator - metabolism Drug Combinations Drug Synergism Epithelial Cells - cytology Epithelial Cells - drug effects Epithelial Cells - metabolism Female High content screening Humans Indoles - pharmacology Infant iPSC-derived CF and CFTR gene-corrected bronchosperes Male microRNA MicroRNAs - genetics Middle Aged Models, Biological Nanoparticles nebulised PLGA nanoparticles Oligonucleotides - genetics Oligonucleotides - pharmacology Original Polylactic Acid-Polyglycolic Acid Copolymer - chemistry Primary bronchial epithelial cells Quinolones - pharmacology RNA sequencing target site blocker
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