Article

<i>In vivo</i> correction of cystic fibrosis mediated by PNA nanoparticles

2022-01-28

Abstract excerpt

Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. We sought to correct the multiple organ dysfunction of the F508del CF-causing mutation using systemic delivery of peptide nucleic acid gene editing technology mediated by biocompatible polymeric nanoparticles. We confirmed phenotypic and genotypic modification in vitro in primary nasal epithelial ce...

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Identifiers and source

Literature Corpus work: f4911996-dd34-50dd-bf6d-a63c2635463e
DOI: 10.1101/2022.01.28.478191

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