Peer-reviewed veterinary case report
Systemic Administered mRNA as Therapy for Metabolic Diseases.
- Journal:
- Trends in molecular medicine
- Year:
- 2019
- Authors:
- Puy, Hervé et al.
- Affiliation:
- UMRs 1149 · France
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.
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Search related cases →Original publication: https://pubmed.ncbi.nlm.nih.gov/30528119/