[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
Abstract 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
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
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