AU2014236156C1 - Compositions and methods for modulating Tau expression - Google Patents
Compositions and methods for modulating Tau expression Download PDFInfo
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- AU2014236156C1 AU2014236156C1 AU2014236156A AU2014236156A AU2014236156C1 AU 2014236156 C1 AU2014236156 C1 AU 2014236156C1 AU 2014236156 A AU2014236156 A AU 2014236156A AU 2014236156 A AU2014236156 A AU 2014236156A AU 2014236156 C1 AU2014236156 C1 AU 2014236156C1
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- compound
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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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- 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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- C12N2310/00—Structure or type of the nucleic acid
- 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
- C12N2310/31—Chemical structure of the backbone
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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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- 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
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
- C12N2310/3181—Peptide nucleic acid, PNA
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- 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
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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
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- 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
- C12N2310/322—2'-R Modification
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- 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
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- 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
- C12N2310/3233—Morpholino-type ring
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- 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
- C12N2310/33—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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
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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
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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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)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Epidemiology (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361785177P | 2013-03-14 | 2013-03-14 | |
| US61/785,177 | 2013-03-14 | ||
| US201461943931P | 2014-02-24 | 2014-02-24 | |
| US61/943,931 | 2014-02-24 | ||
| PCT/US2014/029752 WO2014153236A1 (en) | 2013-03-14 | 2014-03-14 | Compositions and methods for modulating tau expression |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| AU2014236156A1 AU2014236156A1 (en) | 2015-09-24 |
| AU2014236156B2 AU2014236156B2 (en) | 2020-05-28 |
| AU2014236156C1 true AU2014236156C1 (en) | 2020-12-17 |
Family
ID=51581461
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2014236156A Active AU2014236156C1 (en) | 2013-03-14 | 2014-03-14 | Compositions and methods for modulating Tau expression |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US9644207B2 (enExample) |
| EP (2) | EP3766974A1 (enExample) |
| JP (2) | JP6629716B2 (enExample) |
| KR (1) | KR20150130430A (enExample) |
| CN (2) | CN105264091B (enExample) |
| AU (1) | AU2014236156C1 (enExample) |
| BR (1) | BR112015022156A2 (enExample) |
| CA (1) | CA2906119A1 (enExample) |
| ES (1) | ES2807379T3 (enExample) |
| IL (1) | IL241439B (enExample) |
| MX (1) | MX2015012621A (enExample) |
| RU (1) | RU2745324C2 (enExample) |
| WO (1) | WO2014153236A1 (enExample) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013148260A1 (en) | 2012-03-30 | 2013-10-03 | Washington University | Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome |
| ES2807379T3 (es) | 2013-03-14 | 2021-02-22 | Ionis Pharmaceuticals Inc | Composiciones y métodos para regular la expresión de Tau |
| TW202246503A (zh) | 2013-07-19 | 2022-12-01 | 美商百健Ma公司 | 用於調節τ蛋白表現之組合物 |
| PE20171766A1 (es) * | 2015-02-04 | 2017-12-21 | Hoffmann La Roche | Oligomeros antisentido de tau y usos de estos |
| WO2016127000A1 (en) | 2015-02-04 | 2016-08-11 | Bristol-Myers Squibb Company | Methods of selecting therapeutic molecules |
| WO2016151523A1 (en) * | 2015-03-25 | 2016-09-29 | Università Degli Studi Di Trento | Rna interference mediated therapy for neurodegenerative diseases |
| US10370667B2 (en) | 2015-11-18 | 2019-08-06 | Rosalind Franklin University Of Medicine And Science | Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease |
| WO2017087282A1 (en) | 2015-11-18 | 2017-05-26 | Rosalind Franklin University Of Medicine And Science | Antisense compounds targeting leucine-rich repeat kinase 2 (lrrk2) for the treatment of parkinsons disease |
| JOP20200228A1 (ar) * | 2015-12-21 | 2017-06-16 | Novartis Ag | تركيبات وطرق لخفض تعبير البروتين tau |
| JOP20190065A1 (ar) | 2016-09-29 | 2019-03-28 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن tau |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| MX2019006426A (es) * | 2016-12-01 | 2019-08-14 | Sangamo Therapeutics Inc | Reguladores de tau, y composiciones y metodos para su administracion. |
| AU2019233612A1 (en) | 2018-03-13 | 2020-09-10 | Janssen Pharmaceutica Nv | Modified oligonucleotides for use in treatment of tauopathies |
| MY206038A (en) | 2018-07-03 | 2024-11-26 | Hoffmann La Roche | Oligonucleotides for modulating tau expression |
| JP7340794B2 (ja) * | 2018-07-04 | 2023-09-08 | 学校法人 愛知医科大学 | タウのスプライシングを制御するアンチセンスオリゴヌクレオチド及びその用途 |
| CN113226333A (zh) | 2018-10-02 | 2021-08-06 | 桑格摩生物治疗股份有限公司 | 用于调控Tau蛋白的方法和组合物 |
| MY205095A (en) * | 2018-11-21 | 2024-10-01 | Ionis Pharmaceuticals Inc | Compounds and methods for reducing prion expression |
| MX2021014868A (es) * | 2019-06-03 | 2022-03-25 | Quralis Corp | Oligonucleotidos y metodos de uso para el tratamiento de enfermedades neurologicas. |
| AU2021246024A1 (en) * | 2020-03-30 | 2022-10-27 | Alnylam Pharmaceuticals, Inc. | Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof |
| EP4373938A2 (en) * | 2021-07-23 | 2024-05-29 | University Of Kentucky Research Foundation | Inhibition of neurofibrillary tangles using oligonucleotides against circular rnas from the microtubule associated protein tau (mapt) locus |
| US11761284B2 (en) | 2021-07-26 | 2023-09-19 | Benton Frederick Baugh | Method for BOP stack structure |
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| ES2807379T3 (es) | 2021-02-22 |
| EP2971142A4 (en) | 2016-10-19 |
| JP2016513976A (ja) | 2016-05-19 |
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| CN111254145A (zh) | 2020-06-09 |
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| CA2906119A1 (en) | 2014-09-25 |
| US20220243200A1 (en) | 2022-08-04 |
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| US11155815B2 (en) | 2021-10-26 |
| EP2971142A1 (en) | 2016-01-20 |
| WO2014153236A1 (en) | 2014-09-25 |
| JP2019141097A (ja) | 2019-08-29 |
| CN105264091B (zh) | 2020-02-28 |
| RU2015143286A (ru) | 2017-04-17 |
| IL241439B (en) | 2020-03-31 |
| EP2971142B1 (en) | 2020-06-24 |
| JP6629716B2 (ja) | 2020-01-15 |
| US20160032285A1 (en) | 2016-02-04 |
| EP3766974A1 (en) | 2021-01-20 |
| AU2014236156A1 (en) | 2015-09-24 |
| BR112015022156A2 (pt) | 2017-11-14 |
| RU2745324C2 (ru) | 2021-03-23 |
| MX2015012621A (es) | 2016-05-31 |
| CN105264091A (zh) | 2016-01-20 |
| US9644207B2 (en) | 2017-05-09 |
| HK1218659A1 (en) | 2017-03-03 |
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