Skip to main content
. Author manuscript; available in PMC: 2022 Nov 16.
Published in final edited form as: Cell Rep. 2017 Dec 19;21(12):3548–3558. doi: 10.1016/j.celrep.2017.11.081

Figure 1. Strategy of hMUT mRNA Therapy.

Figure 1.

Chemically modified and codon-optimized hMUT mRNA encapsulated in LNP is administrated using an i.v. injection. The LNP delivers the mRNA into hepatocytes where it is translated, undergoes mitochondrial importation and processing, and then forms a homodimer. The MUT enzyme requires the cofactor adenosylcobalamin (AdoCbl) for activity to catalyze the isomerization of L-methylmalonyl-CoA into succinyl-CoA.