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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    • 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
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    • 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
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    • 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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    • C12N2310/00—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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    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—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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  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (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 (enExample)
EP (4) EP4458361A3 (enExample)
JP (1) JP6294876B2 (enExample)
AU (1) AU2013280474B2 (enExample)
CA (1) CA2877905A1 (enExample)
DK (2) DK3461895T3 (enExample)
ES (3) ES2809199T3 (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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