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Systemic Administered mRNA as Therapy for Metabolic Diseases

Hervé Puy1, Jean-Charles Deybach1, Laurent Gouya1

  • 1UMRs 1149, Centre de Recherche sur l'Inflammation, Institut National de la Santé et de la Recherche Médicale, Université Paris Diderot, F-75018 Paris, France; Assistance Publique-Hôpitaux de Paris, HUPNVS Centre Français des Porphyries, Hôpital Louis Mourier, 178 Rue des Renouillers, F-92701 Colombes, France; Laboratory of Excellence Gr-Ex, 75015 Paris, France.

Trends in Molecular Medicine
|December 12, 2018
PubMed
Summary

Messenger RNA (mRNA) therapy shows promise for treating diseases. Intravenous delivery of human porphobilinogen deaminase (PBGD) mRNA effectively replaced defective PBGD protein in preclinical models of acute intermittent porphyria.

Keywords:
RNAacute intermittent porphyrialivermetabolic disordernanoparticles

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