Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

Summary: Isolated methylmalonic acidemia/aciduria (MMA) is a devastating metabolic disorder with poor outcomes despite current medical treatments. Like other mitochondrial enzymopathies, enzyme replacement therapy (ERT) is not available, and although promising, AAV gene therapy can be limited by pre...

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Main Authors: Ding An, Jessica L. Schneller, Andrea Frassetto, Shi Liang, Xuling Zhu, Ji-Sun Park, Matt Theisen, Sue-Jean Hong, Jenny Zhou, Raj Rajendran, Becca Levy, Rebecca Howell, Gilles Besin, Vladimir Presnyak, Staci Sabnis, Kerry E. Murphy-Benenato, E. Sathyajith Kumarasinghe, Timothy Salerno, Cosmin Mihai, Christine M. Lukacs, Randy J. Chandler, Lin T. Guey, Charles P. Venditti, Paolo G.V. Martini
Format: Article
Language:English
Published: Elsevier 2017-12-01
Series:Cell Reports
Online Access:http://www.sciencedirect.com/science/article/pii/S2211124717317485

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http://www.sciencedirect.com/science/article/pii/S2211124717317485

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