Ontology highlight
ABSTRACT:
SUBMITTER: McNeer NA
PROVIDER: S-EPMC4480796 | biostudies-literature | 2015 Apr
REPOSITORIES: biostudies-literature
Nature communications 20150427
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 in vivo gene delivery and transient gene expression. Here we use triplex-forming peptide nucleic acids and donor DNA in biodegradable polymer nanoparticles to correct F508del. We confirm modification with sequencing and a functional c ...[more]