Ontology highlight
ABSTRACT:
SUBMITTER: Prieve MG
PROVIDER: S-EPMC5910669 | biostudies-literature | 2018 Mar
REPOSITORIES: biostudies-literature
Prieve Mary G MG Harvie Pierrot P Monahan Sean D SD Roy Debashish D Li Allen G AG Blevins Teri L TL Paschal Amber E AE Waldheim Matt M Bell Eric C EC Galperin Anna A Ella-Menye Jean-Rene JR Houston Michael E ME
Molecular therapy : the journal of the American Society of Gene Therapy 20180104 3
We describe a novel, two-nanoparticle mRNA delivery system and show that it is highly effective as a means of intracellular enzyme replacement therapy (i-ERT) using a murine model of ornithine transcarbamylase deficiency (OTCD). Our Hybrid mRNA Technology delivery system (HMT) comprises an inert lipid nanoparticle that protects the mRNA from nucleases in the blood as it distributes to the liver and a polymer micelle that targets hepatocytes and triggers endosomal release of mRNA. This results in ...[more]