Systemic Administered mRNA as Therapy for Metabolic Diseases

Hervé Puy; Jean-Charles Deybach; Laurent Gouya

doi:10.1016/j.molmed.2018.11.003

Systemic Administered mRNA as Therapy for Metabolic Diseases

Trends Mol Med. 2019 Jan;25(1):3-5. doi: 10.1016/j.molmed.2018.11.003. Epub 2018 Dec 6.

Authors

Hervé Puy¹, Jean-Charles Deybach², Laurent Gouya³

Affiliations

¹ UMRs 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. Electronic address: herve.puy@aphp.fr.
² UMRs 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. Electronic address: jc.deybach@me.com.
³ UMRs 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. Electronic address: laurent.gouya@inserm.fr.

PMID: 30528119
DOI: 10.1016/j.molmed.2018.11.003

Abstract

The potential of mRNA to produce therapeutic and protective protein levels is a promising approach for the treatment of a large number of diseases. In a recent study published in Nature Medicine (Published online October 8, 2018. doi.org/10.1038/s41591-018-0199-z), the intravenous delivery of human porphobilinogen deaminase (PBGD) mRNA, targeting the liver, demonstrated its efficacy and safety to replace the defective PBGD protein in preclinical models of acute intermittent porphyria.

Keywords: RNA; acute intermittent porphyria; liver; metabolic disorder; nanoparticles.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Animals
Disease Models, Animal
Liver / metabolism
Metabolic Diseases / genetics
Metabolic Diseases / therapy*
Nanoparticles / chemistry
Porphyria, Acute Intermittent / genetics
Porphyria, Acute Intermittent / therapy*
RNA, Messenger / genetics*

Substances

RNA, Messenger