AU2013280474B2 - Modulation of UBE3A-ATS expression - Google Patents

Modulation of UBE3A-ATS expression Download PDF

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Publication number
AU2013280474B2
AU2013280474B2 AU2013280474A AU2013280474A AU2013280474B2 AU 2013280474 B2 AU2013280474 B2 AU 2013280474B2 AU 2013280474 A AU2013280474 A AU 2013280474A AU 2013280474 A AU2013280474 A AU 2013280474A AU 2013280474 B2 AU2013280474 B2 AU 2013280474B2
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ube3a
antisense
ats
antisense compound
modified
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AU2013280474A1 (en
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Arthur L. Beaudet
Linyan MENG
Frank Rigo
Amanda WARD
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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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • 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
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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
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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
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
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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
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    • 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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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Health & Medical Sciences (AREA)
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  • Biomedical Technology (AREA)
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  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
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  • Molecular Biology (AREA)
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  • Medicinal Chemistry (AREA)
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  • 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)
AU2013280474A 2012-06-25 2013-06-25 Modulation of UBE3A-ATS expression Active AU2013280474B2 (en)

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 (2)

Publication Number Publication Date
AU2013280474A1 AU2013280474A1 (en) 2015-01-22
AU2013280474B2 true AU2013280474B2 (en) 2019-01-03

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AU2013280474A Active AU2013280474B2 (en) 2012-06-25 2013-06-25 Modulation of UBE3A-ATS expression

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US (1) US9617539B2 (https=)
EP (4) EP3770258B1 (https=)
JP (1) JP6294876B2 (https=)
AU (1) AU2013280474B2 (https=)
CA (1) CA2877905A1 (https=)
DK (2) DK3461895T3 (https=)
ES (3) ES2809199T3 (https=)
HU (1) HUE051698T2 (https=)
LT (1) LT3461895T (https=)
PL (1) PL3461895T3 (https=)
PT (1) PT3461895T (https=)
SI (1) SI3461895T1 (https=)
WO (1) WO2014004572A2 (https=)

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HUE067905T2 (hu) 2015-11-12 2024-11-28 Hoffmann La Roche Oligonukleotidok paternális UBE3A expresszió indukálására
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PT3717646T (pt) 2017-12-01 2025-07-28 Texas A & M Univ Sys Tratamento antisense da síndrome de angelman
CN111770758A (zh) * 2018-02-27 2020-10-13 北卡罗来纳-查佩尔山大学 用于治疗Angelman综合征的方法和组合物
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CA3113648A1 (en) 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome
JP7241185B2 (ja) * 2019-01-17 2023-03-16 エフ． ホフマン－ラ ロシュ アーゲー Ｅ３ユビキチンリガーゼ（ｕｂｅ３ａ）のタンパク質標的
FI3947684T3 (fi) * 2019-03-29 2025-05-27 Ionis Pharmaceuticals Inc Yhdisteitä ja menetelmiä ube3a-ats:n moduloimiseksi
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