JP5809146B2 - ハンチンチン発現の修飾 - Google Patents

ハンチンチン発現の修飾 Download PDF

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Publication number
JP5809146B2
JP5809146B2 JP2012528949A JP2012528949A JP5809146B2 JP 5809146 B2 JP5809146 B2 JP 5809146B2 JP 2012528949 A JP2012528949 A JP 2012528949A JP 2012528949 A JP2012528949 A JP 2012528949A JP 5809146 B2 JP5809146 B2 JP 5809146B2
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Prior art keywords
isis
certain embodiments
huntingtin
antisense
compound
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Japanese (ja)
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JP2013504329A (ja
JP2013504329A5 (https=
Inventor
ハン，ジーン
ベネット，シー・フランク
フレアー，スーザン・エム
コーダジーウィクツ，ホリー
スタネック，リサ
クリーブランド，ドン・ダブリュー
シハブッディン，ラミア
チェン，セン・エイチ
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
Isis Pharmaceuticals Inc
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/10—Type of nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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JP2012528949A 2009-09-11 2010-09-10 ハンチンチン発現の修飾 Active JP5809146B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US24185309P 2009-09-11 2009-09-11
US61/241,853 2009-09-11
PCT/US2010/048532 WO2011032045A1 (en) 2009-09-11 2010-09-10 Modulation of huntingtin expression

Related Child Applications (1)

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JP2015178281A Division JP6153578B2 (ja) 2009-09-11 2015-09-10 ハンチンチン発現の修飾

Publications (3)

Publication Number Publication Date
JP2013504329A JP2013504329A (ja) 2013-02-07
JP2013504329A5 JP2013504329A5 (https=) 2015-05-14
JP5809146B2 true JP5809146B2 (ja) 2015-11-10

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JP2012528949A Active JP5809146B2 (ja) 2009-09-11 2010-09-10 ハンチンチン発現の修飾
JP2015178281A Active JP6153578B2 (ja) 2009-09-11 2015-09-10 ハンチンチン発現の修飾
JP2017106418A Pending JP2017184754A (ja) 2009-09-11 2017-05-30 ハンチンチン発現の修飾
JP2018180351A Active JP6704439B2 (ja) 2009-09-11 2018-09-26 ハンチンチン発現の修飾
JP2020083833A Pending JP2020115894A (ja) 2009-09-11 2020-05-12 ハンチンチン発現の修飾

Family Applications After (4)

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JP2015178281A Active JP6153578B2 (ja) 2009-09-11 2015-09-10 ハンチンチン発現の修飾
JP2017106418A Pending JP2017184754A (ja) 2009-09-11 2017-05-30 ハンチンチン発現の修飾
JP2018180351A Active JP6704439B2 (ja) 2009-09-11 2018-09-26 ハンチンチン発現の修飾
JP2020083833A Pending JP2020115894A (ja) 2009-09-11 2020-05-12 ハンチンチン発現の修飾

Country Status (19)

Country Link
US (8) US8906873B2 (https=)
EP (3) EP3626823A1 (https=)
JP (5) JP5809146B2 (https=)
KR (5) KR20170102069A (https=)
CN (3) CN111705058A (https=)
AU (4) AU2010292003B2 (https=)
BR (1) BR112012005448B1 (https=)
CA (2) CA2773886C (https=)
DK (1) DK2475675T3 (https=)
ES (2) ES2615534T3 (https=)
HU (1) HUE033113T2 (https=)
IL (4) IL218482A (https=)
MX (3) MX340397B (https=)
NZ (3) NZ714887A (https=)
PL (1) PL2475675T3 (https=)
PT (1) PT2475675T (https=)
RU (2) RU2562861C2 (https=)
SI (1) SI2475675T1 (https=)
WO (1) WO2011032045A1 (https=)

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