Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2015-12

AUTHORS

Nicole Ali McNeer, Kavitha Anandalingam, Rachel J. Fields, Christina Caputo, Sascha Kopic, Anisha Gupta, Elias Quijano, Lee Polikoff, Yong Kong, Raman Bahal, John P. Geibel, Peter M. Glazer, W. Mark Saltzman, Marie E. Egan

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 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 chloride efflux assay. In vitro correction of chloride efflux occurs in up to 25% of human cells. Deep-sequencing reveals negligible off-target effects in partially homologous sites. Intranasal delivery of nanoparticles in CF mice produces changes in the nasal epithelium potential difference assay, consistent with corrected CFTR function. Also, gene correction is detected in the nasal and lung tissue. This work represents facile genome engineering in vivo with oligonucleotides using a nanoparticle system to achieve clinically relevant levels of gene editing without off-target effects. More... »

PAGES

6952

Identifiers

URI

http://scigraph.springernature.com/pub.10.1038/ncomms7952

DOI

http://dx.doi.org/10.1038/ncomms7952

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1023361139

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/25914116


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