JP2013518603A5 - - Google Patents

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Publication number
JP2013518603A5
JP2013518603A5 JP2012552931A JP2012552931A JP2013518603A5 JP 2013518603 A5 JP2013518603 A5 JP 2013518603A5 JP 2012552931 A JP2012552931 A JP 2012552931A JP 2012552931 A JP2012552931 A JP 2012552931A JP 2013518603 A5 JP2013518603 A5 JP 2013518603A5
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JP
Japan
Prior art keywords
chemically modified
modified oligonucleotide
substituted
independently
disease
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2012552931A
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English (en)
Japanese (ja)
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JP2013518603A (ja
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Publication date
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Priority claimed from PCT/US2011/024099 external-priority patent/WO2011097641A1/en
Publication of JP2013518603A publication Critical patent/JP2013518603A/ja
Publication of JP2013518603A5 publication Critical patent/JP2013518603A5/ja
Pending legal-status Critical Current

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JP2012552931A 2010-02-08 2011-02-08 反復伸張に関連する疾患または病態の治療に有用な方法および組成物 Pending JP2013518603A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US30245410P 2010-02-08 2010-02-08
US30245010P 2010-02-08 2010-02-08
US61/302,450 2010-02-08
US61/302,454 2010-02-08
US40515710P 2010-10-20 2010-10-20
US61/405,157 2010-10-20
PCT/US2011/024099 WO2011097641A1 (en) 2010-02-08 2011-02-08 Methods and compositions useful in treatment of diseases or conditions related to repeat expansion

Publications (2)

Publication Number Publication Date
JP2013518603A JP2013518603A (ja) 2013-05-23
JP2013518603A5 true JP2013518603A5 (enExample) 2014-03-20

Family

ID=44355844

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2012552931A Pending JP2013518603A (ja) 2010-02-08 2011-02-08 反復伸張に関連する疾患または病態の治療に有用な方法および組成物

Country Status (4)

Country Link
US (1) US20130059902A1 (enExample)
EP (1) EP2536738A4 (enExample)
JP (1) JP2013518603A (enExample)
WO (1) WO2011097641A1 (enExample)

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