Single-Stranded Phosphorothioated Regions Enhance Cellular Uptake of Cholesterol-Conjugated siRNA but Not Silencing Efficacy
Ly S, Echeverria D, Sousa J, Khvorova A. Mol Ther Nucleic Acids. 2020 Sep 4;21:991-1005. Pubmed
Investigating the pharmacodynamic durability of GalNAc-siRNA conjugates
Brown CR, Gupta S, Qin J, Racie T, He G, Lentini S, Malone R, Yu M, Matsuda S, Shulga-Morskaya S, Nair AV, Theile CS, Schmidt K, Shahraz A, Goel V, Parmar RG, Zlatev I, Schlegel MK, Nair JK, Jayaraman M, Manoharan M, Brown D, Maier ...
Silencing hepatic MCJ attenuates non-alcoholic fatty liver disease (NAFLD) by increasing mitochondrial fatty acid oxidation.
Barbier-Torres L, Fortner KA, Iruzubieta P, Delgado TC, Giddings E, Chen Y, Champagne D, Fernández-Ramos D, Mestre D, Gomez-Santos B, Varela-Rey M, de Juan VG, Fernández-Tussy P, Zubiete-Franco I, García-Monzón C, González-Rodríguez Á, Oza D, Valença-Pereira F, Fang Q, Crespo J, Aspichueta P, Tremblay F, ...
mRNA structural dynamics shape Argonaute-target interactions.
Ruijtenberg S, Sonneveld S, Cui TJ, Logister I, de Steenwinkel D, Xiao Y, MacRae IJ, Joo C, Tanenbaum ME. Nat Struct Mol Biol. 2020 Jul 13. Pubmed
The chemical structure and phosphorothioate content of hydrophobically modified siRNAs impact extrahepatic distribution and efficacy.
Biscans A, Caiazzi J, Davis S, McHugh N, Sousa J, Khvorova A. Nucleic Acids Res. 2020 Jul 16 Pubmed
Argonaute binding within 3′-untranslated regions poorly predicts gene repression
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MicroRNA-206 Regulates Stress-Provoked Aggressive Behaviors in Post-weaning Social Isolation Mice
Chang CH, Kuek EJW, Su CL, Gean PW. Mol Ther Nucleic Acids. 2020 Jun 5;20:812-822. Pubmed
A systems approach delivers a functional microRNA catalog and expanded targets for seizure suppression in temporal lobe epilepsy
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Emerging siRNA Design Principles and Consequences for Biotransformation and Disposition in Drug Development
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Development of an RNAi therapeutic for alpha-1-antitrypsin liver disease
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