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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Publication - Abstract
Jun 23, 2020
Advanced Functional Materials
H. Tanaka, T. Takahashi, M. Konishi, N. Takata and H. Akita et al.
RNA‐based therapeutics is a promising approach for curing intractable diseases by manipulating various cellular functions. For eliciting RNA (i.e., mRNA and siRNA) functions successfully, the RNA in the extracellular space must be protected and it must be delivered to the cytopla...
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