Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

作者：Ding An, Jessica L. Schneller, Andrea Frassetto, Liang Shi, Xuling Zhu, Ji-Sun Park, Matt Theisen, Sue‐Jean Hong, Jenny Y. Zhou, Rajendran Raj, Becca R. Levy, Rebecca M. Howell, Gilles Besin, Vladimir Presnyak, Staci Sabnis, Kerry E. Murphy-Benenato, E. Sathyajith Kumarasinghe, Timothy Salerno, Cosmin Mihai, Christine Lukacs, Randy J. Chandler, Lin T. Guey, Charles P. Venditti, Paolo G.V. Martini · 发表于：Cell Reports · 年份：2018 · DOI：10.1016/j.celrep.2018.08.049 · 被引用次数：25 · 研究领域：Metabolism and Genetic Disorders、RNA modifications and cancer、Virus-based gene therapy research

(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