2'-OMe-phosphorodithioate modified siRNAs show increased loading into the RISC complex and enhanced anti-tumour activity

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Wu, Sherry Y
Yang, Xianbin
Gharpure, Kshipra M
Hatakeyama, Hiroto
Egli, Martin
McGuire, Michael H
Nagaraja, Archana S
Miyake, Takahito M
Rupaimoole, Rajesha
Pecot, Chad V
Taylor, Morgan
Pradeep, Sunila
Sierant, Malgorzata
Rodriguez-Aguayo, Cristian
Choi, Hyun J
Previs, Rebecca A
Armaiz-Pena, Guillermo N
Huang, Li
Martinez, Carlos
Hassell, Tom
Ivan, Cristina
Sehgal, Vasudha
Singhania, Richa
Han, Hee-Dong
Su, Chang
Kim, Ji Hoon
Dalton, Heather J
Kovvali, Chandra
Keyomarsi, Khandan
McMillan, Nigel AJ
Overwijk, Willem W
Liu, Jinsong
Lee, Ju-Seog
Baggerly, Keith A
Lopez-Berestein, Gabriel
Ram, Prahlad T
Nawrot, Barbara
Sood, Anil K
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2014
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Abstract

Improving small interfering RNA (siRNA) efficacy in target cell populations remains a challenge to its clinical implementation. Here, we report a chemical modification, consisting of phosphorodithioate (PS2) and 2'-O-Methyl (2'-OMe) MePS2 on one nucleotide that significantly enhances potency and resistance to degradation for various siRNAs. We find enhanced potency stems from an unforeseen increase in siRNA loading to the RNA-induced silencing complex, likely due to the unique interaction mediated by 2'-OMe and PS2. We demonstrate the therapeutic utility of MePS2 siRNAs in chemoresistant ovarian cancer mouse models via targeting GRAM domain containing 1B (GRAMD1B), a protein involved in chemoresistance. GRAMD1B silencing is achieved in tumours following MePS2-modified siRNA treatment, leading to a synergistic anti-tumour effect in combination with paclitaxel. Given the previously limited success in enhancing siRNA potency with chemically modified siRNAs, our findings represent an important advance in siRNA design with the potential for application in numerous cancer types.

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Nature Communications

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5

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Biochemistry and cell biology not elsewhere classified

Cancer genetics

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