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Donald J. Foster et al.
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Aaron D. Springer et al.
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Liangliang Zhang et al.
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Matthew L. Hammill et al.
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Wei Jin et al.
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Elad Elkayam et al.
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Anastasia Khvorova et al.
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Kevin Fitzgerald et al.
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Daniel Mutisya et al.
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Xinyun Song et al.
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Ivan V. Chernikov et al.
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Rubina Parmar et al.
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Identification of metabolically stable 5′- phosphate analogs that support single-stranded siRNA activity
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Terminus-free siRNA prepared by photo-crosslinking activated via slicing by Ago2
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Jenny K. W. Lam et al.
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Monica J. Piatek et al.
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Jayaprakash K. Nair et al.
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Single modification at position 14 of siRNA strand abolishes its gene-silencing activity by decreasing both RISC loading and target degradation
Jie Zheng et al.
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Multiple sensors ensure guide strand selection in human RNAi pathways
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Dongbo Yu et al.
CELL (2012)
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Junbin Zhang et al.
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Zhongping Yuan et al.
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Takanori Kubo et al.
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Christian Dohmen et al.
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Amides as Excellent Mimics of Phosphate Linkages in RNA
Chelliah Selvam et al.
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Naoko Abe et al.
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Giulia Cuccato et al.
BMC SYSTEMS BIOLOGY (2011)
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Gabor Butora et al.
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Pooja Dua et al.
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Yuanyu Huang et al.
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Unexpected origins of the enhanced pairing affinity of 2′-fluoro-modified RNA
Pradeep S. Pallan et al.
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Narendra Vaish et al.
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Application of small interfering RNAs modified by unlocked nucleic acid (UNA) to inhibit the heart-pathogenic coxsackievirus B3
Denise Werk et al.
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Punit P. Seth et al.
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Maria B. Laursen et al.
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Fedor V. Karginov et al.
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Filipp Frank et al.
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Sihem Cheloufi et al.
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Denise M. Kenski et al.
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Jun-Xia Wei et al.
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Kazutaka Nishina et al.
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Masayuki Sano et al.
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Solid-phase synthesis of modified RNAs containing amide-linked oligoribonucleosides at their 3 '-end and their application to siRNA
Reiko Iwase et al.
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Samit Shah et al.
OLIGONUCLEOTIDES (2007)
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James O. McNamara et al.
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Widespread siRNA off-target'' transcript silencing mediated by seed region sequence complementarity
Aimee L. Jackson et al.
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A Birmingham et al.
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CR Allerson et al.
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XY Lin et al.
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Locked nucleic acid (LNA) mediated improvements in siRNA stability and functionality
J Elmen et al.
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Argonaute2 is the catalytic engine of mammalian RNAi
JD Liu et al.
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C Lorenz et al.
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