JP2014501507A5 - - Google Patents

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JP2014501507A5
JP2014501507A5 JP2013540028A JP2013540028A JP2014501507A5 JP 2014501507 A5 JP2014501507 A5 JP 2014501507A5 JP 2013540028 A JP2013540028 A JP 2013540028A JP 2013540028 A JP2013540028 A JP 2013540028A JP 2014501507 A5 JP2014501507 A5 JP 2014501507A5
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JP
Japan
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modified oligonucleotide
modified
wing segment
nucleoside
synuclein
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JP2013540028A
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JP2014501507A (ja
JP6126009B2 (ja
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Priority claimed from PCT/US2011/061245 external-priority patent/WO2012068405A2/en
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Publication of JP2014501507A5 publication Critical patent/JP2014501507A5/ja
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JP2013540028A 2010-11-17 2011-11-17 α−シヌクレイン発現の調節 Active JP6126009B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US41484810P 2010-11-17 2010-11-17
US61/414,848 2010-11-17
PCT/US2011/061245 WO2012068405A2 (en) 2010-11-17 2011-11-17 Modulation of alpha synuclein expression

Related Child Applications (1)

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JP2017004099A Division JP2017079786A (ja) 2010-11-17 2017-01-13 α−シヌクレイン発現の調節

Publications (3)

Publication Number Publication Date
JP2014501507A JP2014501507A (ja) 2014-01-23
JP2014501507A5 true JP2014501507A5 (enExample) 2016-07-07
JP6126009B2 JP6126009B2 (ja) 2017-05-10

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JP2013540028A Active JP6126009B2 (ja) 2010-11-17 2011-11-17 α−シヌクレイン発現の調節
JP2017004099A Withdrawn JP2017079786A (ja) 2010-11-17 2017-01-13 α−シヌクレイン発現の調節

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US (4) US9663783B2 (enExample)
EP (2) EP2640853B1 (enExample)
JP (2) JP6126009B2 (enExample)
AU (2) AU2011329777B2 (enExample)
CA (2) CA3077910A1 (enExample)
WO (1) WO2012068405A2 (enExample)

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