Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis

Ema Robinson, Kelvin D. MacDonald, Kai Slaughter, Madison McKinney, Siddharth Patel, Conroy Sun, Gaurav Sahay

Research output: Contribution to journalArticlepeer-review

172 Scopus citations

Abstract

The use of lipid nanoparticles to deliver therapeutic mRNA is an increasingly attractive gene therapy modality. Here, Robinson et al. demonstrate that lipid nanoparticle transfection of mRNA can repair the central defect in cystic fibrosis, chloride transport, both in vitro and in vivo. The authors discuss why this technology is a promising treatment option for monogenic disorders such as cystic fibrosis.

Original languageEnglish (US)
Pages (from-to)2034-2046
Number of pages13
JournalMolecular Therapy
Volume26
Issue number8
DOIs
StatePublished - Aug 1 2018

Keywords

  • cystic fibrosis
  • gene therapy
  • ion transport
  • mRNA therapeutics
  • nanoparticles
  • nasal potential difference

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics
  • Pharmacology
  • Drug Discovery

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