PL3461895T3 - Modulacja ekspresji ube3a-ats - Google Patents
Modulacja ekspresji ube3a-atsInfo
- Publication number
- PL3461895T3 PL3461895T3 PL18188705T PL18188705T PL3461895T3 PL 3461895 T3 PL3461895 T3 PL 3461895T3 PL 18188705 T PL18188705 T PL 18188705T PL 18188705 T PL18188705 T PL 18188705T PL 3461895 T3 PL3461895 T3 PL 3461895T3
- Authority
- PL
- Poland
- Prior art keywords
- ube3a
- modulation
- ats
- expression
- ats expression
- Prior art date
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- 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
- 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
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- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
Landscapes
- 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)
Applications Claiming Priority (8)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201261664083P | 2012-06-25 | 2012-06-25 | |
US201261738959P | 2012-12-18 | 2012-12-18 | |
US201361750939P | 2013-01-10 | 2013-01-10 | |
US201361755617P | 2013-01-23 | 2013-01-23 | |
US201361772925P | 2013-03-05 | 2013-03-05 | |
EP18188705.0A EP3461895B1 (en) | 2012-06-25 | 2013-06-25 | Modulation of ube3a-ats expression |
EP13809694.6A EP2864479B1 (en) | 2012-06-25 | 2013-06-25 | Modulation of ube3a-ats expression |
PCT/US2013/047701 WO2014004572A2 (en) | 2012-06-25 | 2013-06-25 | Modulation of ube3a-ats expression |
Publications (1)
Publication Number | Publication Date |
---|---|
PL3461895T3 true PL3461895T3 (pl) | 2021-01-11 |
Family
ID=49784000
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PL18188705T PL3461895T3 (pl) | 2012-06-25 | 2013-06-25 | Modulacja ekspresji ube3a-ats |
Country Status (13)
Country | Link |
---|---|
US (1) | US9617539B2 (pl) |
EP (3) | EP3461895B1 (pl) |
JP (1) | JP6294876B2 (pl) |
AU (1) | AU2013280474B2 (pl) |
CA (1) | CA2877905A1 (pl) |
DK (2) | DK2864479T3 (pl) |
ES (2) | ES2809199T3 (pl) |
HU (1) | HUE051698T2 (pl) |
LT (1) | LT3461895T (pl) |
PL (1) | PL3461895T3 (pl) |
PT (1) | PT3461895T (pl) |
SI (1) | SI3461895T1 (pl) |
WO (1) | WO2014004572A2 (pl) |
Families Citing this family (17)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2016086104A1 (en) * | 2014-11-25 | 2016-06-02 | Ionis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
EP3291843B1 (en) | 2015-05-07 | 2023-03-22 | University of South Florida | Modified ube3a gene for a gene therapy approach for angelman syndrome |
CN108350431A (zh) * | 2015-11-12 | 2018-07-31 | 豪夫迈·罗氏有限公司 | 用于确定候选药物的功效特征的方法 |
EP3548005A4 (en) | 2016-11-29 | 2020-06-17 | Puretech Health LLC | EXOSOMES FOR THE ADMINISTRATION OF THERAPEUTIC AGENTS |
SG11202003596WA (en) * | 2017-12-01 | 2020-05-28 | Texas A & M Univ Sys | Angelman syndrome antisense treatment |
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 |
JP2021527087A (ja) | 2018-06-14 | 2021-10-11 | オービッド・セラピューティクス・インコーポレイテッドOvid Therapeutics Inc. | アンジェルマン症候群の処置におけるMIR−92aまたはMIR−145の使用 |
MX2021003303A (es) * | 2018-09-21 | 2021-06-23 | Univ Connecticut | Composiciones y metodos para restaurar la expresion del gen ube3a paterno en sindrome de angelman humano. |
US20220091137A1 (en) * | 2019-01-17 | 2022-03-24 | Hoffmann-La Roche Inc. | E3 ubiquitin ligase (ube3a) protein targets |
JP2022527105A (ja) * | 2019-03-29 | 2022-05-30 | 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 | Ube3a-atsを調節するための化合物及び方法 |
CA3139678A1 (en) * | 2019-05-22 | 2020-11-26 | The University Of North Carolina At Chapel Hill | Ube3a genes and expression cassettes and their use |
US20220265607A1 (en) * | 2019-07-16 | 2022-08-25 | The University Of North Carolina At Chapel Hill | Methods and compositions for unsilencing imprinted genes |
US20230042176A1 (en) * | 2019-12-18 | 2023-02-09 | National University Of Singapore | Method for treating angelman syndrome and related disorders |
WO2021222232A1 (en) * | 2020-04-28 | 2021-11-04 | The Trustees Of The University Of Pennsylvania | Compositions and uses thereof for treatment of angelman syndrome |
WO2022169861A2 (en) * | 2021-02-03 | 2022-08-11 | The Board Of Regents Of The University Of Texas System | Gene therapy for angelman syndrome |
US20240240179A1 (en) * | 2021-05-05 | 2024-07-18 | The General Hospital Corporation | Therapy for treatment of prader-willi syndrome |
WO2023168000A1 (en) * | 2022-03-03 | 2023-09-07 | The Regents Of The University Of California | Compositions and methods for treatment of angelman syndrome |
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-
2013
- 2013-06-25 SI SI201331770T patent/SI3461895T1/sl unknown
- 2013-06-25 ES ES18188705T patent/ES2809199T3/es active Active
- 2013-06-25 EP EP18188705.0A patent/EP3461895B1/en not_active Revoked
- 2013-06-25 HU HUE18188705A patent/HUE051698T2/hu unknown
- 2013-06-25 PL PL18188705T patent/PL3461895T3/pl unknown
- 2013-06-25 LT LTEP18188705.0T patent/LT3461895T/lt unknown
- 2013-06-25 DK DK13809694.6T patent/DK2864479T3/en active
- 2013-06-25 DK DK18188705.0T patent/DK3461895T3/da active
- 2013-06-25 AU AU2013280474A patent/AU2013280474B2/en active Active
- 2013-06-25 CA CA2877905A patent/CA2877905A1/en active Pending
- 2013-06-25 EP EP20182895.1A patent/EP3770258B1/en active Active
- 2013-06-25 PT PT181887050T patent/PT3461895T/pt unknown
- 2013-06-25 US US14/409,348 patent/US9617539B2/en active Active
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- 2013-06-25 ES ES13809694.6T patent/ES2688831T3/es active Active
- 2013-06-25 EP EP13809694.6A patent/EP2864479B1/en active Active
- 2013-06-25 JP JP2015520410A patent/JP6294876B2/ja active Active
Also Published As
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EP3461895B1 (en) | 2020-07-01 |
WO2014004572A2 (en) | 2014-01-03 |
EP2864479A2 (en) | 2015-04-29 |
DK3461895T3 (da) | 2020-07-20 |
AU2013280474B2 (en) | 2019-01-03 |
WO2014004572A3 (en) | 2014-05-01 |
CA2877905A1 (en) | 2014-01-03 |
EP3770258B1 (en) | 2024-07-17 |
JP6294876B2 (ja) | 2018-03-14 |
DK2864479T3 (en) | 2018-10-22 |
EP3461895A1 (en) | 2019-04-03 |
AU2013280474A1 (en) | 2015-01-22 |
SI3461895T1 (sl) | 2020-10-30 |
PT3461895T (pt) | 2020-09-08 |
HUE051698T2 (hu) | 2021-03-29 |
ES2688831T3 (es) | 2018-11-07 |
EP2864479A4 (en) | 2016-06-08 |
LT3461895T (lt) | 2020-09-10 |
US9617539B2 (en) | 2017-04-11 |
US20150191723A1 (en) | 2015-07-09 |
EP3770258A1 (en) | 2021-01-27 |
ES2809199T3 (es) | 2021-03-03 |
EP2864479B1 (en) | 2018-08-15 |
JP2015529635A (ja) | 2015-10-08 |
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