JP2015511821A - Ａｔｘｎ３発現の調節のための組成物および方法 - Google Patents

Ａｔｘｎ３発現の調節のための組成物および方法 Download PDF

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JP2015511821A
JP2015511821A JP2015500521A JP2015500521A JP2015511821A JP 2015511821 A JP2015511821 A JP 2015511821A JP 2015500521 A JP2015500521 A JP 2015500521A JP 2015500521 A JP2015500521 A JP 2015500521A JP 2015511821 A JP2015511821 A JP 2015511821A
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oligonucleotide
atxn3
seq
expression
nucleotide
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JP2015511821A5 (enExample
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ナタリー・ウスカテギ
マイ・ヘドチェーン
イェンス・ボー・ローデ・ハンセン
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Roche Innovation Center Copenhagen AS
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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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    • 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
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    • 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
    • C12N15/1137—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 against enzymes
    • A—HUMAN NECESSITIES
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2015500521A 2012-03-12 2013-03-12 Ａｔｘｎ３発現の調節のための組成物および方法 Pending JP2015511821A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261609774P 2012-03-12 2012-03-12
US61/609,774 2012-03-12
PCT/US2013/030553 WO2013138353A2 (en) 2012-03-12 2013-03-12 Compositions and methods for modulation of atxn3 expression

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JP2015511821A true JP2015511821A (ja) 2015-04-23
JP2015511821A5 JP2015511821A5 (enExample) 2015-11-12

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US (1) US20150315595A1 (enExample)
EP (1) EP2839008A4 (enExample)
JP (1) JP2015511821A (enExample)
CN (1) CN104254610A (enExample)
BR (1) BR112014021612A2 (enExample)
HK (1) HK1204652A1 (enExample)
WO (1) WO2013138353A2 (enExample)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021522800A (ja) * 2018-05-09 2021-09-02 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Ａｔｘｎ３発現を低減するための化合物及び方法
JP2021530973A (ja) * 2019-06-06 2021-11-18 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft Ａｔｘｎ３を標的とするアンチセンスオリゴヌクレオチド
JPWO2021230286A1 (enExample) * 2020-05-12 2021-11-18

Families Citing this family (13)

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EP3353303B1 (en) * 2015-09-25 2023-08-02 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
JOP20190104A1 (ar) * 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
WO2019233921A1 (en) 2018-06-05 2019-12-12 F. Hoffmann-La Roche Ag Oligonucleotides for modulating atxn2 expression
JP2022507283A (ja) * 2018-11-19 2022-01-18 ユニキュアー アイピー ビー．ブイ． 脊髄小脳失調症３型の治療のためのアタキシン－３のｒｎａｉ誘導低減
MX2021008331A (es) * 2019-01-09 2021-08-05 Univ De Coimbra Arn bicatenario y usos del mismo.
JP2022520986A (ja) * 2019-02-22 2022-04-04 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ａｔｘｎ３発現を減少させるための化合物及び方法
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
US20230056569A1 (en) * 2019-11-22 2023-02-23 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
US20230060373A1 (en) 2020-12-03 2023-03-02 Hoffmann-La Roche Inc. Antisense Oligonucleotides Targeting ATXN3
US20220177883A1 (en) 2020-12-03 2022-06-09 Hoffmann-La Roche Inc. Antisense Oligonucleotides Targeting ATXN3
WO2023052317A1 (en) 2021-09-29 2023-04-06 F. Hoffmann-La Roche Ag Rna editing
CN116064667B (zh) * 2022-12-09 2024-07-02 中南大学湘雅医院 基于CRISPR/Cas9的人源化ATXN3基因敲入小鼠模型的构建方法及应用
WO2025252669A1 (en) 2024-06-03 2025-12-11 Evotec International Gmbh Modified oligonucleotides for reducing atxn3 expression

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US20040241854A1 (en) * 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
JP2005304321A (ja) * 2004-04-19 2005-11-04 Japan Science & Technology Agency 変異ＭＪＤ遺伝子の発現を特異的に抑制するｓｉＲＮＡ
JP2006518197A (ja) * 2003-02-10 2006-08-10 サンタリス・ファルマ・アクティーゼルスカブ ラス発現の改変のためのオリゴマー化合物
JP2010507579A (ja) * 2006-10-18 2010-03-11 アイシス ファーマシューティカルズ， インコーポレーテッド アンチセンス化合物
WO2010120508A2 (en) * 2009-03-31 2010-10-21 The General Hospital Corporation Regulation of mir-33 micrornas in the treatment of cholesterol-related disorders

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JP2011516489A (ja) * 2008-03-31 2011-05-26 ユニバーシティ・オブ・サウス・フロリダ 疾患誘発性運動失調症および非運動失調性平衡異常の治療法
AU2009276763B2 (en) * 2008-07-29 2015-07-16 The Board Of Regents Of The University Of Texas Sytem Selective inhibition of polyglutamine protein expression
EP3561060A1 (en) * 2010-02-08 2019-10-30 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
US9161948B2 (en) * 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates

Patent Citations (5)

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US20040241854A1 (en) * 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
JP2006518197A (ja) * 2003-02-10 2006-08-10 サンタリス・ファルマ・アクティーゼルスカブ ラス発現の改変のためのオリゴマー化合物
JP2005304321A (ja) * 2004-04-19 2005-11-04 Japan Science & Technology Agency 変異ＭＪＤ遺伝子の発現を特異的に抑制するｓｉＲＮＡ
JP2010507579A (ja) * 2006-10-18 2010-03-11 アイシス ファーマシューティカルズ， インコーポレーテッド アンチセンス化合物
WO2010120508A2 (en) * 2009-03-31 2010-10-21 The General Hospital Corporation Regulation of mir-33 micrornas in the treatment of cholesterol-related disorders

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BMC MOLECULAR BIOLOGY, vol. 13, no. 6, JPN6016023641, 7 March 2012 (2012-03-07), pages 1 - 12, ISSN: 0003342750 *
GENE THERAPY, vol. 18, JPN6016023644, 2011, pages 326 - 333, ISSN: 0003342751 *
NUCLEIC ACIDS RESEARCH, vol. 38, no. 1, JPN6016023650, 2010, pages 3, ISSN: 0003342753 *
PLOS ONE, vol. 5, no. 5, JPN6016023646, 2010, pages 10682, ISSN: 0003342752 *

Cited By (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021522800A (ja) * 2018-05-09 2021-09-02 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Ａｔｘｎ３発現を低減するための化合物及び方法
JP2021530973A (ja) * 2019-06-06 2021-11-18 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft Ａｔｘｎ３を標的とするアンチセンスオリゴヌクレオチド
JP7155302B2 (ja) 2019-06-06 2022-10-18 エフ．ホフマン－ラ ロシュ アーゲー Ａｔｘｎ３を標的とするアンチセンスオリゴヌクレオチド
US11542501B2 (en) 2019-06-06 2023-01-03 Hoffmann-La Roche Inc. Antisense oligonucleotides targeting ATXN3
JPWO2021230286A1 (enExample) * 2020-05-12 2021-11-18
WO2021230286A1 (ja) * 2020-05-12 2021-11-18 田辺三菱製薬株式会社 Ａｔａｘｉｎ ３発現を調節するための化合物、方法及び医薬組成物
EP4151237A4 (en) * 2020-05-12 2024-08-14 Mitsubishi Tanabe Pharma Corporation Compound, method and pharmaceutical composition for regulating expression of ataxin 3
JP7776420B2 (ja) 2020-05-12 2025-11-26 田辺三菱製薬株式会社 Ａｔａｘｉｎ ３発現を調節するための化合物、方法及び医薬組成物

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Publication number Publication date
US20150315595A1 (en) 2015-11-05
EP2839008A4 (en) 2015-12-02
BR112014021612A2 (pt) 2017-07-18
WO2013138353A3 (en) 2013-11-14
EP2839008A2 (en) 2015-02-25
WO2013138353A2 (en) 2013-09-19
HK1204652A1 (en) 2015-11-27
CN104254610A (zh) 2014-12-31

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