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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JP2013504329A5 (https=
JP2013504329A (ja
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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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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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    • C12N15/09—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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    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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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
    • 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
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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) EP3178933B1 (https=)
JP (5) JP5809146B2 (https=)
KR (5) KR20170102069A (https=)
CN (3) CN104894129B (https=)
AU (4) AU2010292003B2 (https=)
BR (1) BR112012005448B1 (https=)
CA (2) CA2773886C (https=)
DK (1) DK2475675T3 (https=)
ES (2) ES2772825T3 (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) RU2751847C9 (https=)
SI (1) SI2475675T1 (https=)
WO (1) WO2011032045A1 (https=)

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