[HTML] Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium
NA McNeer, K Anandalingam, RJ Fields… - Nature …, 2015 - nature.com
Cystic fibrosis (CF) is a lethal genetic disorder most commonly caused by the F508del
mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It is not
readily amenable to gene therapy because of its systemic nature and challenges including …
mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It is not
readily amenable to gene therapy because of its systemic nature and challenges including …
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