JP6294876B2 - Ｕｂｅ３ａ−ａｔｓ発現の調節 - Google Patents

Ｕｂｅ３ａ−ａｔｓ発現の調節 Download PDF

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Publication number
JP6294876B2
JP6294876B2 JP2015520410A JP2015520410A JP6294876B2 JP 6294876 B2 JP6294876 B2 JP 6294876B2 JP 2015520410 A JP2015520410 A JP 2015520410A JP 2015520410 A JP2015520410 A JP 2015520410A JP 6294876 B2 JP6294876 B2 JP 6294876B2
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ube3a
ats
region
nucleic acid
modified
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JP2015529635A5 (enExample
JP2015529635A (ja
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リゴ，フランク
ワード，アマンダ
ボーデ，アーサー・エル
メン，リンヤン
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Baylor College of Medicine
Ionis Pharmaceuticals Inc
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Baylor College of Medicine
Ionis Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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
    • C—CHEMISTRY; METALLURGY
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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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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2015520410A 2012-06-25 2013-06-25 Ｕｂｅ３ａ−ａｔｓ発現の調節 Active JP6294876B2 (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201261664083P 2012-06-25 2012-06-25
US61/664,083 2012-06-25
US201261738959P 2012-12-18 2012-12-18
US61/738,959 2012-12-18
US201361750939P 2013-01-10 2013-01-10
US61/750,939 2013-01-10
US201361755617P 2013-01-23 2013-01-23
US61/755,617 2013-01-23
US201361772925P 2013-03-05 2013-03-05
US61/772,925 2013-03-05
PCT/US2013/047701 WO2014004572A2 (en) 2012-06-25 2013-06-25 Modulation of ube3a-ats expression

Publications (3)

Publication Number Publication Date
JP2015529635A JP2015529635A (ja) 2015-10-08
JP2015529635A5 JP2015529635A5 (enExample) 2016-08-12
JP6294876B2 true JP6294876B2 (ja) 2018-03-14

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Family Applications (1)

Application Number Title Priority Date Filing Date
JP2015520410A Active JP6294876B2 (ja) 2012-06-25 2013-06-25 Ｕｂｅ３ａ−ａｔｓ発現の調節

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US (1) US9617539B2 (enExample)
EP (4) EP3770258B1 (enExample)
JP (1) JP6294876B2 (enExample)
AU (1) AU2013280474B2 (enExample)
CA (1) CA2877905A1 (enExample)
DK (2) DK2864479T3 (enExample)
ES (3) ES2987225T3 (enExample)
HU (1) HUE051698T2 (enExample)
LT (1) LT3461895T (enExample)
PL (1) PL3461895T3 (enExample)
PT (1) PT3461895T (enExample)
SI (1) SI3461895T1 (enExample)
WO (1) WO2014004572A2 (enExample)

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JP6841507B2 (ja) 2015-05-07 2021-03-10 ユニヴァーシティ オブ サウス フロリダ アンジェルマン症候群の遺伝子治療法のための組み換えｕｂｅ３ａ遺伝子
KR102840652B1 (ko) 2015-11-12 2025-08-04 에프. 호프만-라 로슈 아게 부계 ube3a 발현을 유도하기 위한 올리고뉴클레오티드
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JP2021513866A (ja) * 2018-02-27 2021-06-03 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル アンジェルマン症候群を処置する方法および組成物
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