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...
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 |
Similar Items
-
Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemiaResearch in context
by: Ding An, et al.
Published: (2019-07-01) -
Methylmalonic Acidemia and Extrapyramidal Disease
by: J Gordon Millichap
Published: (1988-11-01) -
Methylmalonic acidemia with emergency hypertension
by: Bahareh Yaghmaei, et al.
Published: (2016-01-01) -
Methylmalonic acidemia with emergency hypertension
by: Bahareh Yaghmaei, et al.
Published: (2016-01-01) -
The Value of Liver Transplantation for Methylmalonic Acidemia
by: Yi-Zhou Jiang, et al.
Published: (2019-03-01)