Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

(Cell Reports 21, 3548–3558; December 19, 2017) In the originally published version of this article, there were two typos when referring to the chemistry of the mRNA used. In the following sentence, 5-methoxy UTP was mistakenly written as 1-methylpseudoUTP. The correct sentence is shown here and now appears with the article online. “Briefly, mRNA was synthesized in vitro by T7 RNA polymerase-mediated transcription where the UTP was substituted with 5-methoxy UTP.” In the following sentence, 5-methoxyU was mistakenly written as pseudoU. The correct sentence is shown here and now appears with the article online. “To develop a new class of therapy for MMA, we generated a 5-methoxyU-modified codon-optimized messenger RNA (mRNA) encoding human methylmalonyl-CoA mutase (hMUT).” The authors apologize for the error. Systemic Messenger RNA Therapy as a Treatment for Methylmalonic AcidemiaAn et al.Cell ReportsDecember 19, 2017In BriefAn et al. find that systemically delivered LNP-encapsulated mRNA results in hepatic protein expression. hMUT mRNA expresses functional mitochondrial MUT enzyme, and MMA mouse models show a metabolic and clinical response after mRNA therapy. Full-Text PDF Open Access