MX2016004651A - Composiciones para modular la expresion de c9orf72. - Google Patents
Composiciones para modular la expresion de c9orf72.Info
- Publication number
- MX2016004651A MX2016004651A MX2016004651A MX2016004651A MX2016004651A MX 2016004651 A MX2016004651 A MX 2016004651A MX 2016004651 A MX2016004651 A MX 2016004651A MX 2016004651 A MX2016004651 A MX 2016004651A MX 2016004651 A MX2016004651 A MX 2016004651A
- Authority
- MX
- Mexico
- Prior art keywords
- c90rf72
- compositions
- methods
- mrna
- exon
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 3
- 108020004999 messenger RNA Proteins 0.000 abstract 3
- 230000000692 anti-sense effect Effects 0.000 abstract 2
- 150000001875 compounds Chemical class 0.000 abstract 2
- 239000003112 inhibitor Substances 0.000 abstract 2
- 230000002401 inhibitory effect Effects 0.000 abstract 1
- 230000004770 neurodegeneration Effects 0.000 abstract 1
- 208000015122 neurodegenerative disease Diseases 0.000 abstract 1
- 230000001717 pathogenic effect Effects 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- 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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- 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
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
- G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
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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
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
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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
- C12N2310/31—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
- 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
- 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
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Hematology (AREA)
- Urology & Nephrology (AREA)
- Immunology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Analytical Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pathology (AREA)
- Cell Biology (AREA)
- General Physics & Mathematics (AREA)
- Food Science & Technology (AREA)
- Psychiatry (AREA)
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361890108P | 2013-10-11 | 2013-10-11 | |
| US201361891313P | 2013-10-15 | 2013-10-15 | |
| US201361919540P | 2013-12-20 | 2013-12-20 | |
| US201461927903P | 2014-01-15 | 2014-01-15 | |
| US201461980502P | 2014-04-16 | 2014-04-16 | |
| PCT/US2014/060194 WO2015054676A2 (en) | 2013-10-11 | 2014-10-11 | Compositions for modulating c9orf72 expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2016004651A true MX2016004651A (es) | 2016-08-05 |
Family
ID=52813758
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2016004651A MX2016004651A (es) | 2013-10-11 | 2014-10-11 | Composiciones para modular la expresion de c9orf72. |
| MX2018010676A MX2018010676A (es) | 2013-10-11 | 2016-04-11 | Composiciones para modular la expresion de c9orf72. |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2018010676A MX2018010676A (es) | 2013-10-11 | 2016-04-11 | Composiciones para modular la expresion de c9orf72. |
Country Status (16)
| Country | Link |
|---|---|
| US (3) | US10221414B2 (enExample) |
| EP (2) | EP4166667A3 (enExample) |
| JP (2) | JP6679476B2 (enExample) |
| KR (1) | KR20160062069A (enExample) |
| CN (2) | CN105637090B (enExample) |
| AU (2) | AU2014331652B2 (enExample) |
| BR (1) | BR112016007751A2 (enExample) |
| CA (1) | CA2926408A1 (enExample) |
| CL (3) | CL2016000839A1 (enExample) |
| IL (1) | IL244759A0 (enExample) |
| MX (2) | MX2016004651A (enExample) |
| MY (1) | MY192689A (enExample) |
| PH (1) | PH12016500603A1 (enExample) |
| RU (1) | RU2748426C2 (enExample) |
| SG (2) | SG10201808903UA (enExample) |
| WO (1) | WO2015054676A2 (enExample) |
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| SG171914A1 (en) | 2008-12-02 | 2011-07-28 | Chiralgen Ltd | Method for the synthesis of phosphorus atom modified nucleic acids |
| IN2012DN00720A (enExample) | 2009-07-06 | 2015-06-19 | Ontorii Inc | |
| WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
| MX347361B (es) | 2011-07-19 | 2017-04-12 | Wave Life Sciences Ltd | Metodos para la sintesis de acidos nucleicos funcionalizados. |
| EP2872485B1 (en) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| WO2014062736A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for monitoring c9orf72 expression |
| WO2014062686A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for modulating c9orf72 expression |
| AU2013331434B2 (en) | 2012-10-15 | 2019-08-08 | Ionis Pharmaceuticals, Inc. | Compositions for modulating C90RF72 expression |
| RU2748426C2 (ru) | 2013-10-11 | 2021-05-25 | Ионис Фармасьютикалз, Инк. | Композиции для модуляции экспрессии c90rf72 |
| CN106068325B (zh) | 2014-01-16 | 2021-07-09 | 波涛生命科学有限公司 | 手性设计 |
| EP4410805A3 (en) | 2014-03-18 | 2024-11-27 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| EP3722424A1 (en) | 2015-04-16 | 2020-10-14 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| BR112017028194B1 (pt) | 2015-07-10 | 2023-03-14 | Ionis Pharmaceuticals, Inc | Composto oligomérico modulador de diaciglicerol aciltransferase 2 (dgat2), composição, e seus usos |
| US11260073B2 (en) | 2015-11-02 | 2022-03-01 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating C90RF72 |
| CN105663129B (zh) * | 2015-12-29 | 2020-05-15 | 山东大学 | 用于治疗肌萎缩侧索硬化和额颞叶痴呆的化合物与应用 |
| CA3017532A1 (en) | 2016-04-13 | 2017-10-19 | Ionis Pharmaceuticals, Inc. | Methods for reducing c9orf72 expression |
| CN110177544A (zh) | 2016-11-29 | 2019-08-27 | 普尔泰克健康有限公司 | 用于递送治疗剂的外泌体 |
| EP3622073A4 (en) | 2017-05-09 | 2021-01-06 | University of Massachusetts | Methods of treating amyotrophic lateral sclerosis (als) |
| JP2020534253A (ja) | 2017-08-08 | 2020-11-26 | ウェーブ ライフ サイエンシーズ リミテッド | オリゴヌクレオチド組成物及びその方法 |
| WO2019060686A1 (en) | 2017-09-22 | 2019-03-28 | University Of Massachusetts | NEW DUAL EXPRESSION VECTORS OF SOD1 AND USES THEREOF |
| US20210261981A1 (en) * | 2017-10-23 | 2021-08-26 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| US10905707B2 (en) * | 2017-11-10 | 2021-02-02 | University Of Massachusetts | Compositions and methods for the treatment of expanded repeat-associated disorders |
| AU2019208011B2 (en) | 2018-01-12 | 2025-06-05 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| WO2019140231A1 (en) | 2018-01-12 | 2019-07-18 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| EP3849564A4 (en) * | 2018-09-10 | 2023-08-09 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating cln3 expression |
| WO2020072428A1 (en) * | 2018-10-01 | 2020-04-09 | United Neuroscience | Peptide immunogen constructs directed against dipeptide repeat proteins from c9orf72 |
| CA3121749A1 (en) | 2018-12-06 | 2020-06-11 | Biogen Ma Inc. | Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis |
| US20220380773A1 (en) * | 2019-01-29 | 2022-12-01 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing app expression |
| WO2020205605A2 (en) * | 2019-03-29 | 2020-10-08 | University Of Massachusetts | OLIGONUCLEOTIDE-BASED MODULATION OF C9orf72 |
| CN114072501A (zh) | 2019-05-06 | 2022-02-18 | 马萨诸塞大学 | 抗c9orf72寡核苷酸及相关方法 |
| CN115151641A (zh) * | 2019-12-13 | 2022-10-04 | 阿尔尼拉姆医药品有限公司 | 人类染色体9开放阅读框72(C9ORF72)iRNA剂组合物及其使用方法 |
| WO2021159008A2 (en) | 2020-02-07 | 2021-08-12 | Maze Therapeutics, Inc. | Compositions and methods for treating neurodegenerative diseases |
| EP4110924A4 (en) * | 2020-02-28 | 2024-03-13 | Ionis Pharmaceuticals, Inc. | COMPOUNDS AND METHODS FOR MODULATING PRE-MRNA SPLICEING |
| EP4125930A4 (en) * | 2020-03-27 | 2025-06-04 | University Of Massachusetts | Dual-acting siRNA-based modulation of C9ORF72 |
| EP4133073A2 (en) * | 2020-04-09 | 2023-02-15 | Association Institut de Myologie | Antisense sequences for treating amyotrophic lateral sclerosis |
| UY39344A (es) | 2020-07-28 | 2022-02-25 | Ionis Pharmaceuticals Inc | Compuestos y métodos para reducir la expresión de app |
| MX2023011794A (es) | 2021-04-06 | 2024-01-08 | Maze Therapeutics Inc | Composiciones y métodos para el tratamiento de la proteinopatía de proteina de union a acido desoxirribonucleico tar 43 (tdp-43). |
| JP2025535055A (ja) | 2022-10-05 | 2025-10-22 | トレース ニューロサイエンス， インコーポレイテッド | Ｕｎｃ１３ａアンチセンスオリゴヌクレオチド及びその使用 |
| WO2025107038A1 (en) * | 2023-11-23 | 2025-05-30 | Murdoch University | Method of treating motor neurone disease |
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