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. Author manuscript; available in PMC: 2017 Jan 1.
Published in final edited form as: Expert Opin Ther Pat. 2016;26(3):323–345. doi: 10.1517/13543776.2016.1154045

Figure 18.

Figure 18

The ASO “5–10–5” design of ISIS416858. It is a 20–base pair ASO designed to support RNaseH activity. The 2‘-methoxyethylribose is utilized only on the external 5 nucleotides on both ends while leaving the internal 10 nucleotides with the unmodified 2‘-deoxyribose. The phosphorothioate backbone is used along the length of the ASO to provide nuclease resistance. This provides increased target RNA binding affinity on the outer portions of the ASO, while still allowing RNaseH cleavage at the central domain of the ASO.