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

    64 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

    Fingerprint

    Dive into the research topics of 'Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis'. Together they form a unique fingerprint.

    Cite this