Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain

The use of siRNA-based therapies for the treatment of neurodegenerative disease requires efficient, nontoxic distribution to the affected brain parenchyma, notably the striatum and cortex. Here, we describe the synthesis and activity of a fully chemically modified siRNA that is directly conjugated t...

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Bibliografiske detaljer
Udgivet i:Mol Ther Nucleic Acids
Main Authors: Nikan, Mehran, Osborn, Maire F, Coles, Andrew H, Godinho, Bruno MDC, Hall, Lauren M, Haraszti, Reka A, Hassler, Matthew R, Echeverria, Dimas, Aronin, Neil, Khvorova, Anastasia
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2016
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5023396/
https://ncbi.nlm.nih.gov/pubmed/27504598
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2016.50
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