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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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主要な著者: Mehran Nikan, Maire F Osborn, Andrew H Coles, Bruno MDC Godinho, Lauren M Hall, Reka A Haraszti, Matthew R Hassler, Dimas Echeverria, Neil Aronin, Anastasia Khvorova
フォーマット: Artigo
言語:Inglês
出版事項: Elsevier 2016-01-01
シリーズ:Molecular Therapy: Nucleic Acids
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オンライン・アクセス:http://www.sciencedirect.com/science/article/pii/S2162253117300677
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