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

• Main Authors: Mehran Nikan (Author), Maire F Osborn (Author), Andrew H Coles (Author), Bruno MDC Godinho (Author), Lauren M Hall (Author), Reka A Haraszti (Author), Matthew R Hassler (Author), Dimas Echeverria (Author), Neil Aronin (Author), Anastasia Khvorova (Author)
• Format: Book
• Published: Elsevier, 2016-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...