MX2017008500A - Supresión del gen de la huntingtina inducida por la arni. - Google Patents
Supresión del gen de la huntingtina inducida por la arni.Info
- Publication number
- MX2017008500A MX2017008500A MX2017008500A MX2017008500A MX2017008500A MX 2017008500 A MX2017008500 A MX 2017008500A MX 2017008500 A MX2017008500 A MX 2017008500A MX 2017008500 A MX2017008500 A MX 2017008500A MX 2017008500 A MX2017008500 A MX 2017008500A
- Authority
- MX
- Mexico
- Prior art keywords
- rna
- huntingtine
- arni
- suppression
- double stranded
- 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
- 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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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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
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2310/3519—Fusion with another nucleic acid
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Neurosurgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Psychology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
La presente invención provee un ARN de doble cadena que comprende una primera secuencia de ARN y una segunda secuencia de ARN, en donde la primera y segunda secuencias de ARN son sustancialmente complementarias, en donde la primera secuencia de ARN tiene una longitud de secuencia de por lo menos 19 nucleótidos y es sustancialmente complementaria con SEQ ID NO. 1; dicho ARN de doble cadena es para uso de la inducción de ARNi contra las secuencias del exón 1 de la Huntingtina; el ARN de doble cadena de conformidad con la invención fue capaz de reducir la muerte de las células neuronales de la huntingtina en un modelo animal.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP14200308 | 2014-12-24 | ||
| PCT/EP2015/081157 WO2016102664A1 (en) | 2014-12-24 | 2015-12-23 | Rnai induced huntingtin gene suppression |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| MX2017008500A true MX2017008500A (es) | 2018-02-01 |
| MX381121B MX381121B (es) | 2025-03-12 |
Family
ID=52282540
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2017008500A MX381121B (es) | 2014-12-24 | 2015-12-23 | Supresión del gen de la huntingtina inducida por la arni |
Country Status (25)
| Country | Link |
|---|---|
| US (4) | US10174321B2 (es) |
| EP (2) | EP3237618B1 (es) |
| JP (2) | JP6839094B2 (es) |
| KR (1) | KR102636633B1 (es) |
| CN (1) | CN108064292B (es) |
| AU (1) | AU2015370903B2 (es) |
| CA (1) | CA2971920C (es) |
| CO (1) | CO2017007335A2 (es) |
| CY (1) | CY1121733T1 (es) |
| DK (1) | DK3237618T3 (es) |
| EA (1) | EA037696B1 (es) |
| ES (1) | ES2732023T3 (es) |
| HK (1) | HK1246344B (es) |
| HR (1) | HRP20190992T1 (es) |
| HU (1) | HUE043842T2 (es) |
| IL (1) | IL252990B (es) |
| LT (1) | LT3237618T (es) |
| MX (1) | MX381121B (es) |
| PL (1) | PL3237618T3 (es) |
| PT (1) | PT3237618T (es) |
| RS (1) | RS58862B1 (es) |
| SI (1) | SI3237618T1 (es) |
| SM (1) | SMT201900347T1 (es) |
| TR (1) | TR201908969T4 (es) |
| WO (1) | WO2016102664A1 (es) |
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| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| BR112014001244A2 (pt) | 2011-07-19 | 2017-02-21 | Wave Life Sciences Pte Ltd | métodos para a síntese de ácidos nucléicos funcionalizados |
| CN112007045A (zh) | 2012-07-13 | 2020-12-01 | 波涛生命科学有限公司 | 手性控制 |
| ES2862073T3 (es) | 2012-07-13 | 2021-10-06 | Wave Life Sciences Ltd | Grupo auxiliar asimétrico |
| BR112015000723A2 (pt) | 2012-07-13 | 2017-06-27 | Shin Nippon Biomedical Laboratories Ltd | adjuvante de ácido nucléico quiral |
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| JP6892433B2 (ja) | 2015-04-03 | 2021-06-23 | ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ | 十分に安定化された非対称ｓｉｒｎａ |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
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| CA3024448C (en) | 2016-05-18 | 2025-09-09 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| BR112018073472A2 (pt) | 2016-05-18 | 2019-08-27 | Voyager Therapeutics Inc | composições e métodos de tratamento da doença de huntington |
| US10457940B2 (en) * | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
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| KR20200033249A (ko) | 2017-06-05 | 2020-03-27 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료 화합물 |
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| WO2011158924A1 (ja) * | 2010-06-18 | 2011-12-22 | 独立行政法人国立精神・神経医療研究センター | 優性アレル発現抑制剤 |
| EP2990477B8 (en) | 2011-09-08 | 2018-10-31 | uniQure IP B.V. | Removal of contaminating viruses from aav preparations |
| CN103224556B (zh) * | 2013-01-31 | 2015-04-15 | 清华大学 | 亨廷顿舞蹈症的靶位蛋白质及其编码基因和应用 |
| EA037696B1 (ru) * | 2014-12-24 | 2021-05-12 | ЮНИКЕР АйПи Б.В. | Супрессия гена гентингтина, индуцированная рнк-интерференцией |
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