%0 Journal Article
%T Systemic Administered mRNA as Therapy for Metabolic Diseases
%J -
%D 2019
%R https://doi.org/10.1016/j.molmed.2018.11.003
%X 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
%U https://www.cell.com/trends/molecular-medicine/fulltext/S1471-4914(18)30220-X