Figure 4.

mRNA-based approach for enzyme replacement therapy. In general, mouse models for metabolic disorders from the liver constitute enzyme-deficient mice characterized by the accumulation of toxic serum concentrations of metabolites. mRNA-LNPs inoculated i.m. or i.v. are transported via the bloodstream to the liver where the message is translated, and the protein is synthesized by hepatocytes. Newly produced protein replaces the deficient or aberrant protein associated to the disease resulting in the rescue of metabolic function and fast reduction of toxic metabolite levels, consequently generating a therapeutic effect. Figure was created with biorender.com (accessed on 19 August 2022).