CN104254610A - 调节atxn3表达的组合物和方法 - Google Patents

调节atxn3表达的组合物和方法 Download PDF

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Publication number
CN104254610A
CN104254610A CN201380013752.5A CN201380013752A CN104254610A CN 104254610 A CN104254610 A CN 104254610A CN 201380013752 A CN201380013752 A CN 201380013752A CN 104254610 A CN104254610 A CN 104254610A
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China
Prior art keywords
oligonucleotide
atxn3
seq
expression
nucleic acid
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Pending
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CN201380013752.5A
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English (en)
Chinese (zh)
Inventor
N·乌斯卡特吉
M·黑特耶恩
J·B·R·海森
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Enzon Pharmaceuticals Inc
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Enzon Pharmaceuticals Inc
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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
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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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
    • C—CHEMISTRY; METALLURGY
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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
    • 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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  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN201380013752.5A 2012-03-12 2013-03-12 调节atxn3表达的组合物和方法 Pending CN104254610A (zh)

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

Publications (1)

Publication Number Publication Date
CN104254610A true CN104254610A (zh) 2014-12-31

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CN201380013752.5A Pending CN104254610A (zh) 2012-03-12 2013-03-12 调节atxn3表达的组合物和方法

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Country Link
US (1) US20150315595A1 (https=)
EP (1) EP2839008A4 (https=)
JP (1) JP2015511821A (https=)
CN (1) CN104254610A (https=)
BR (1) BR112014021612A2 (https=)
HK (1) HK1204652A1 (https=)
WO (1) WO2013138353A2 (https=)

Cited By (3)

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Publication number Priority date Publication date Assignee Title
CN112189053A (zh) * 2018-05-09 2021-01-05 Ionis制药公司 用于减少atxn3表达的化合物和方法
CN113302302A (zh) * 2019-01-09 2021-08-24 科英布拉大学 双链rna及其用途
CN113383077A (zh) * 2018-11-19 2021-09-10 优尼科Ip有限公司 RNAi诱导的ataxin-3减少以用于治疗脊髓小脑共济失调3型

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EP4285912A3 (en) * 2015-09-25 2024-07-10 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
JP7379387B2 (ja) 2018-06-05 2023-11-14 エフ． ホフマン－ラ ロシュ アーゲー Ａｔｘｎ２発現を制御するためのオリゴヌクレオチド
WO2020172559A1 (en) * 2019-02-22 2020-08-27 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
CN113950529A (zh) * 2019-06-06 2022-01-18 豪夫迈·罗氏有限公司 靶向atxn3的反义寡核苷酸
US12565653B2 (en) 2019-11-22 2026-03-03 Alnylam Pharmaceuticals, Inc. ATAXIN3 (ATXN3) RNAi agent compositions and methods of use thereof
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
AR124229A1 (es) 2020-12-03 2023-03-01 Hoffmann La Roche Oligonucleótidos antisentido que actúan sobre atxn3
US20230060373A1 (en) 2020-12-03 2023-03-02 Hoffmann-La Roche Inc. Antisense Oligonucleotides Targeting ATXN3
JP2024536132A (ja) 2021-09-29 2024-10-04 エフ． ホフマン－ラ ロシュ アーゲー Ｒｎａ編集方法
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
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
JP4463608B2 (ja) * 2004-04-19 2010-05-19 独立行政法人科学技術振興機構 変異ＭＪＤ遺伝子の発現を特異的に抑制するｓｉＲＮＡ
ES2526295T5 (es) * 2006-10-18 2021-05-04 Ionis Pharmaceuticals Inc Compuestos antisentido
WO2009146031A1 (en) * 2008-03-31 2009-12-03 University Of South Florida Methods of treating disease-induced ataxia and non-ataxic imbalance
CA2732343C (en) * 2008-07-29 2017-05-09 The Board Of Regents Of The University Of Texas System Selective inhibition of polyglutamine protein expression
EP2414521B1 (en) * 2009-03-31 2016-10-26 The General Hospital Corporation Regulation of mir-33 micrornas in the treatment of cholesterol-related disorders
US8957040B2 (en) * 2010-02-08 2015-02-17 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
US9161948B2 (en) * 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates

Non-Patent Citations (2)

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FISZER.ET AL: "An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases", 《BMC MOLECULAR BIOLOGY》 *
VICTOR M.MILLER ET AL: "ALLELE-SPECIFIC SILENCING OF DOMINANT DISEASE GENES.", 《PNAS》 *

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112189053A (zh) * 2018-05-09 2021-01-05 Ionis制药公司 用于减少atxn3表达的化合物和方法
CN112189053B (zh) * 2018-05-09 2024-05-14 Ionis制药公司 用于减少atxn3表达的化合物和方法
CN113383077A (zh) * 2018-11-19 2021-09-10 优尼科Ip有限公司 RNAi诱导的ataxin-3减少以用于治疗脊髓小脑共济失调3型
CN113302302A (zh) * 2019-01-09 2021-08-24 科英布拉大学 双链rna及其用途

Also Published As

Publication number Publication date
JP2015511821A (ja) 2015-04-23
WO2013138353A3 (en) 2013-11-14
BR112014021612A2 (pt) 2017-07-18
EP2839008A2 (en) 2015-02-25
HK1204652A1 (en) 2015-11-27
EP2839008A4 (en) 2015-12-02
US20150315595A1 (en) 2015-11-05
WO2013138353A2 (en) 2013-09-19

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