JP2016526529A5 - - Google Patents

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Publication number
JP2016526529A5
JP2016526529A5 JP2015560881A JP2015560881A JP2016526529A5 JP 2016526529 A5 JP2016526529 A5 JP 2016526529A5 JP 2015560881 A JP2015560881 A JP 2015560881A JP 2015560881 A JP2015560881 A JP 2015560881A JP 2016526529 A5 JP2016526529 A5 JP 2016526529A5
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JP
Japan
Prior art keywords
nucleotides
strand
independently selected
nucleotide analogs
natural
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JP2015560881A
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English (en)
Japanese (ja)
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JP2016526529A (ja
JP6604544B2 (ja
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Priority claimed from PCT/JP2014/003208 external-priority patent/WO2014203518A1/en
Publication of JP2016526529A publication Critical patent/JP2016526529A/ja
Publication of JP2016526529A5 publication Critical patent/JP2016526529A5/ja
Application granted granted Critical
Publication of JP6604544B2 publication Critical patent/JP6604544B2/ja
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JP2015560881A 2013-06-16 2014-06-16 エクソンスキッピング効果を有する二本鎖アンチセンス核酸 Active JP6604544B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361835634P 2013-06-16 2013-06-16
US61/835,634 2013-06-16
US201361836672P 2013-06-19 2013-06-19
US61/836,672 2013-06-19
PCT/JP2014/003208 WO2014203518A1 (en) 2013-06-16 2014-06-16 Double-stranded antisense nucleic acid with exon-skipping effect

Publications (3)

Publication Number Publication Date
JP2016526529A JP2016526529A (ja) 2016-09-05
JP2016526529A5 true JP2016526529A5 (enExample) 2017-08-17
JP6604544B2 JP6604544B2 (ja) 2019-11-13

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Family Applications (1)

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JP2015560881A Active JP6604544B2 (ja) 2013-06-16 2014-06-16 エクソンスキッピング効果を有する二本鎖アンチセンス核酸

Country Status (7)

Country Link
US (1) US10190117B2 (enExample)
EP (1) EP3010514B1 (enExample)
JP (1) JP6604544B2 (enExample)
CN (1) CN105324119A (enExample)
AU (1) AU2014282666A1 (enExample)
CA (1) CA2915443A1 (enExample)
WO (1) WO2014203518A1 (enExample)

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