Publication - Summary
Sep 24, 2018
Nucleic Acid Therapeutics
R. Jain, J.P. Frederick, E.Y. Huang, K.E. Burke, D.M. Mauger, E.A. Andrianova, S.J. Farlow, S. Siddiqui, J. Pimentel, K. Cheung-Ong, K.M. McKinney, C. Köhrer, M.J. Moore and T. Chakraborty
The advent of therapeutic mRNAs significantly increases the possibilities of protein-based biologics beyond those that can be synthesized by recombinant technologies (eg, monoclonal antibodies, extracellular enzymes, and cytokines). In addition to their application in the areas o...
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Publication - Summary
Dec 19, 2017
Cell Reports
D. An, J.L. Schneller, A. Frassetto, S. Liang, X. Zhu, J.S. Park, M. Theisen, S.J. Hong, J. Zhou J, R. Rajendran, B. Levy, R. Howell, G. Besin, V. Presnyak, S. Sabnis, K.E. Murphy-Benenato, E.S. Kumarasinghe, T. Salerno and P.G.V. Martini PGV
Inborn errors of metabolism (IEMs) can be relatively straight forward to screen for and diagnose; these disorders however, have currently very limited options for treatment. Methylmalonic acidemia (MMA), is an IEM caused by complete or partial defi...
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