Overcoming GNA/RNA base-pairing limitations using isonucleotides improves the pharmacodynamic activity of ESC+ GalNAc-siRNAs

We recently reported that RNAi-mediated off-target effects are important drivers of the hepatotoxicity observed for a subset of GalNAc–siRNA conjugates in rodents, and that these findings could be mitigated by seed-pairing destabilization using a single GNA nucleotide placed within the seed region o...

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Detalles Bibliográficos
Autores principales: Schlegel, Mark K, Matsuda, Shigeo, Brown, Christopher R, Harp, Joel M, Barry, Joseph D, Berman, Daniel, Castoreno, Adam, Schofield, Sally, Szeto, John, Manoharan, Muthiah, Charissé, Klaus, Egli, Martin, Maier, Martin A
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2021
Materias:
Chemical Biology and Nucleic Acid Chemistry
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8565336/
https://www.ncbi.nlm.nih.gov/pubmed/34648028
http://dx.doi.org/10.1093/nar/gkab916

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8565336/
https://www.ncbi.nlm.nih.gov/pubmed/34648028
http://dx.doi.org/10.1093/nar/gkab916

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