MX2017013199A - Composiciones para modular la expresion de c90rf72. - Google Patents
Composiciones para modular la expresion de c90rf72.Info
- Publication number
- MX2017013199A MX2017013199A MX2017013199A MX2017013199A MX2017013199A MX 2017013199 A MX2017013199 A MX 2017013199A MX 2017013199 A MX2017013199 A MX 2017013199A MX 2017013199 A MX2017013199 A MX 2017013199A MX 2017013199 A MX2017013199 A MX 2017013199A
- Authority
- MX
- Mexico
- Prior art keywords
- compositions
- c9orf72 expression
- modulating
- modulating c9orf72
- methods
- Prior art date
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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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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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
- A61P25/22—Anxiolytics
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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
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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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
- C12N2310/10—Type of nucleic acid
- 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
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- 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
- C12N2310/322—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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- 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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- 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
- C12N2310/341—Gapmers, i.e. of the type ===---===
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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
- C12N2310/30—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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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- 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
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- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Neurology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Genetics & Genomics (AREA)
- Neurosurgery (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Psychology (AREA)
- Pain & Pain Management (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)
- Saccharide Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Abstract
En la presente se describen composiciones y métodos para reducir la expresión de proteína y ARNm de C9ORF72 en un animal. Estos métodos son útiles para tratar, prevenir, mejorar o enlentecer el avance de enfermedades neurodegenerativas en un individuo que lo necesite.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562148691P | 2015-04-16 | 2015-04-16 | |
US201562232941P | 2015-09-25 | 2015-09-25 | |
US201562239400P | 2015-10-09 | 2015-10-09 | |
PCT/US2016/027747 WO2016168592A2 (en) | 2015-04-16 | 2016-04-15 | Compositions for modulating c9orf72 expression |
Publications (2)
Publication Number | Publication Date |
---|---|
MX2017013199A true MX2017013199A (es) | 2018-02-09 |
MX366128B MX366128B (es) | 2019-06-28 |
Family
ID=57126261
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2017013199A MX366128B (es) | 2015-04-16 | 2016-04-15 | Composiciones para modular la expresion de c90rf72. |
MX2019007834A MX2019007834A (es) | 2015-04-16 | 2017-10-12 | Composiciones para modular la expresion de c9orf72. |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2019007834A MX2019007834A (es) | 2015-04-16 | 2017-10-12 | Composiciones para modular la expresion de c9orf72. |
Country Status (30)
Country | Link |
---|---|
US (4) | US9605263B2 (es) |
EP (2) | EP3283080B1 (es) |
JP (3) | JP6606557B2 (es) |
KR (2) | KR102104163B1 (es) |
CN (2) | CN113265404A (es) |
AU (2) | AU2016249002B2 (es) |
BR (1) | BR112017016068A2 (es) |
CA (1) | CA2978103A1 (es) |
CL (4) | CL2017002567A1 (es) |
CO (1) | CO2017007671A2 (es) |
CY (1) | CY1122981T1 (es) |
DK (1) | DK3283080T3 (es) |
ES (1) | ES2791995T3 (es) |
HK (1) | HK1250255A1 (es) |
HR (1) | HRP20200843T1 (es) |
HU (1) | HUE049233T2 (es) |
IL (2) | IL253557B (es) |
LT (1) | LT3283080T (es) |
MX (2) | MX366128B (es) |
MY (1) | MY181815A (es) |
NZ (1) | NZ733882A (es) |
PH (2) | PH12017501875B1 (es) |
PL (1) | PL3283080T3 (es) |
PT (1) | PT3283080T (es) |
RS (1) | RS60230B1 (es) |
RU (1) | RU2736574C2 (es) |
SA (3) | SA517390168B1 (es) |
SG (2) | SG10202001856WA (es) |
SI (1) | SI3283080T1 (es) |
WO (1) | WO2016168592A2 (es) |
Families Citing this family (23)
Publication number | Priority date | Publication date | Assignee | Title |
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EP2370451B1 (en) | 2008-12-02 | 2016-11-16 | Wave Life Sciences Japan, Inc. | Method for the synthesis of phosphorus atom modified nucleic acids |
KR101885383B1 (ko) | 2009-07-06 | 2018-08-03 | 웨이브 라이프 사이언시스 리미티드 | 신규한 핵산 프로드러그 및 그의 사용 방법 |
US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
AU2012284265B2 (en) | 2011-07-19 | 2017-08-17 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
WO2014062686A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for modulating c9orf72 expression |
EP2906697A4 (en) | 2012-10-15 | 2016-06-22 | Ionis Pharmaceuticals Inc | METHODS OF MONITORING C9ORF72 EXPRESSION |
BR112015008399A8 (pt) | 2012-10-15 | 2017-10-03 | Ionis Pharmaceuticals Inc | Composto para modular a expressão de c90rf72, seu uso, oligonucleotídeo modificado, composto de fita dupla e composição |
CA2926408A1 (en) | 2013-10-11 | 2015-04-16 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
SG10201912897UA (en) | 2014-01-16 | 2020-02-27 | Wave Life Sciences Ltd | Chiral design |
WO2017079291A1 (en) | 2015-11-02 | 2017-05-11 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating c90rf72 |
AU2017248637A1 (en) * | 2016-04-13 | 2018-09-27 | Ionis Pharmaceuticals, Inc. | Methods for reducing C9ORF72 expression |
US11795453B2 (en) * | 2016-10-31 | 2023-10-24 | Emendobio, Inc. | Compositions for genome editing |
AU2018316159A1 (en) * | 2017-08-08 | 2020-02-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
US10905707B2 (en) | 2017-11-10 | 2021-02-02 | University Of Massachusetts | Compositions and methods for the treatment of expanded repeat-associated disorders |
JP7455746B2 (ja) | 2018-01-12 | 2024-03-26 | ブリストル-マイヤーズ スクイブ カンパニー | アルファ-シヌクレインを標的とするアンチセンスオリゴヌクレオチドおよびその使用 |
CA3107352A1 (en) * | 2018-07-23 | 2020-01-30 | Enclear Therapies, Inc. | Methods of treating neurological disorders |
WO2020027311A1 (ja) | 2018-08-03 | 2020-02-06 | 国立大学法人東京医科歯科大学 | Tdp-43凝集阻害剤 |
US20220034907A1 (en) | 2018-12-06 | 2022-02-03 | Biogen Ma Inc. | Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis |
AU2020252020A1 (en) * | 2019-03-29 | 2021-11-11 | University Of Massachusetts | Oligonucleotide-based modulation of C90RF72 |
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 |
IL291974A (en) * | 2019-10-06 | 2022-06-01 | Wave Life Sciences Ltd | Compositions of oligonucleotides and methods of using them |
WO2024036186A2 (en) * | 2022-08-09 | 2024-02-15 | The University Of Massachusetts | Identification of allele-specific target sequences for c9orf72 |
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