HRP20190992T1 - Suprimiranje gena za huntingtin inducirano s rnai - Google Patents
Suprimiranje gena za huntingtin inducirano s rnai Download PDFInfo
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- HRP20190992T1 HRP20190992T1 HRP20190992TT HRP20190992T HRP20190992T1 HR P20190992 T1 HRP20190992 T1 HR P20190992T1 HR P20190992T T HRP20190992T T HR P20190992TT HR P20190992 T HRP20190992 T HR P20190992T HR P20190992 T1 HRP20190992 T1 HR P20190992T1
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- Prior art keywords
- seq
- double
- stranded rna
- expression cassette
- rna
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- 108090000623 proteins and genes Proteins 0.000 title claims 2
- 108091030071 RNAI Proteins 0.000 title 1
- 230000001629 suppression Effects 0.000 title 1
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 15
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims 13
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 6
- 238000001415 gene therapy Methods 0.000 claims 5
- 239000013598 vector Substances 0.000 claims 5
- 108020004414 DNA Proteins 0.000 claims 4
- 239000002679 microRNA Substances 0.000 claims 4
- 239000013607 AAV vector Substances 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- 208000023105 Huntington disease Diseases 0.000 claims 1
- 108091033773 MiR-155 Proteins 0.000 claims 1
- 108091027967 Small hairpin RNA Proteins 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 claims 1
- 108091032770 miR-451 stem-loop Proteins 0.000 claims 1
- 108091030646 miR-451a stem-loop Proteins 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 108091007428 primary miRNA Proteins 0.000 claims 1
- 239000004055 small Interfering RNA Substances 0.000 claims 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
- A61K31/713—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 N.A.
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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 N.A.
- C12N2310/141—MicroRNAs, miRNAs
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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
- C12N2310/3519—Fusion with another nucleic acid
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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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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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
- C12N2320/32—Special delivery means, e.g. tissue-specific
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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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
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- 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)
Claims (14)
1. Dvolančana RNA koja sadrži prvi RNA slijed i drugi RNA slijed, gdje su prvi i drugi RNA slijed uglavnom komplementarni, naznačena time što prvi RNA slijed ima duljinu slijeda od najmanje 19 nukleotida i komplementaran je SEQ ID NO. 1, gdje se prvi RNA slijed bira iz skupine koju čine SEQ ID NO.3, SEQ ID NO.4, SEQ ID NO.5, SEQ ID NO.6, te SEQ ID NO.7, te gdje navedena dvolančana RNA može smanjiti eksprimiranje gena za huntingtin.
2. Dvolančana RNA u skladu s patentnim zahtjevom 1, naznačena time što dvolančanu RNA sadrži pre-miRNA okosnica, pri-miRNA okosnica, shRNA ili siRNA, po mogućnosti pre-miRNA okosnica.
3. Dvolančana RNA u skladu s bilo kojim od patentnih zahtjeva 1 ili patentnim zahtjevom 2, naznačena time što se prvi RNA slijed i drugi RNA slijed bira iz skupine koju čine kombinacije SEQ ID NO. 3 i 8; SEQ ID NO. 4 i 9; SEQ ID NO. 5 i 10; SEQ ID NO. 5 i 13; SEQ ID NO. 5 i 14; SEQ ID NO. 6 i 11; i SEQ ID NO. 7 i 12.
4. Dvolančana RNA u skladu s bilo kojim od patentnih zahtjeva 1-3, naznačena time što dvolančanu RNA sadrži pre-miRNA okosnica od miR-451a ili miR-155.
5. Dvolančana RNA u skladu s patentnim zahtjevom 4, naznačena time što dvolančanu RNA sadrži RNA koja sadrži SEQ ID NO. 15, SEQ ID NO. 16, SEQ ID NO. 20, SEQ ID NO. 21 ili SEQ ID NO. 22.
6. DNA, naznačena time što kodira dvolančanu RNA u skladu s bilo kojim od patentnih zahtjeva 1-5.
7. Ekspresijska kaseta, naznačena time što kodira dvolančanu RNA u skladu s bilo kojim od patentnih zahtjeva 1-5.
8. Ekspresijska kaseta u skladu s patentnim zahtjevom 7, naznačena time što ekspresijska kaseta sadrži PGK promotor, CMV promotor, neurospecifični promotor ili CBA promotor.
9. Ekspresijska kaseta u skladu s patentnim zahtjevom 8, naznačena time što ekspresijska kaseta sadrži SEQ ID NO. 17, 18 ili 19.
10. Vektor za gensku terapiju, naznačen time što sadrži ekspresijsku kasetu u skladu s bilo kojim od patentnih zahtjeva 7-9.
11. Vektor za gensku terapiju u skladu s patentnim zahtjevom 10, naznačen time što je vektor za gensku terapiju AAV vektor, po mogućnosti AAV vektor serotipa 5.
12. Stanica domaćin, naznačena time što sadrži DNA u skladu s patentnim zahtjevom 6 ili ekspresijsku kasetu u skladu s bilo kojim od patentnih zahtjeva 7-9.
13. Dvolančana RNA u skladu s bilo kojim od patentnih zahtjeva 1-5, DNA u skladu s patentnim zahtjevom 6, ekspresijska kaseta u skladu s bilo kojim od patentnih zahtjeva 7-9, vektor za gensku terapiju u skladu s patentnim zahtjevom 10 ili patentnim zahtjevom 11, naznačeni time što su namijenjeni upotrebi u medicinskom liječenju.
14. Dvolančana RNA u skladu s bilo kojim od patentnih zahtjeva 1-5, DNA u skladu s patentnim zahtjevom 6, ekspresijska kaseta u skladu s bilo kojim od patentnih zahtjeva 7-9, vektor za gensku terapiju u skladu s patentnim zahtjevom 10 ili patentnim zahtjevom 11, naznačeni time što su namijenjeni upotrebi u liječenju Huntingtonove bolesti.
Applications Claiming Priority (3)
| 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 |
| EP15817385.6A EP3237618B1 (en) | 2014-12-24 | 2015-12-23 | Rnai induced huntingtin gene suppression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20190992T1 true HRP20190992T1 (hr) | 2019-09-20 |
Family
ID=52282540
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20190992TT HRP20190992T1 (hr) | 2014-12-24 | 2019-05-31 | Suprimiranje gena za huntingtin inducirano s rnai |
Country Status (24)
| Country | Link |
|---|---|
| US (4) | US10174321B2 (hr) |
| EP (2) | EP3237618B1 (hr) |
| JP (2) | JP6839094B2 (hr) |
| KR (1) | KR102636633B1 (hr) |
| CN (1) | CN108064292B (hr) |
| AU (1) | AU2015370903B2 (hr) |
| CA (1) | CA2971920C (hr) |
| CO (1) | CO2017007335A2 (hr) |
| CY (1) | CY1121733T1 (hr) |
| DK (1) | DK3237618T3 (hr) |
| EA (1) | EA037696B1 (hr) |
| ES (1) | ES2732023T3 (hr) |
| HK (1) | HK1246344B (hr) |
| HR (1) | HRP20190992T1 (hr) |
| HU (1) | HUE043842T2 (hr) |
| IL (1) | IL252990B (hr) |
| LT (1) | LT3237618T (hr) |
| MX (1) | MX2017008500A (hr) |
| PL (1) | PL3237618T3 (hr) |
| PT (1) | PT3237618T (hr) |
| RS (1) | RS58862B1 (hr) |
| SI (1) | SI3237618T1 (hr) |
| TR (1) | TR201908969T4 (hr) |
| WO (1) | WO2016102664A1 (hr) |
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| EP2620428B1 (en) | 2010-09-24 | 2019-05-22 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| SG10201700554VA (en) | 2011-07-19 | 2017-03-30 | Wave Life Sciences Pte Ltd | Methods for the synthesis of functionalized nucleic acids |
| SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| BR112015000723A2 (pt) | 2012-07-13 | 2017-06-27 | Shin Nippon Biomedical Laboratories Ltd | adjuvante de ácido nucléico quiral |
| WO2014012081A2 (en) | 2012-07-13 | 2014-01-16 | Ontorii, Inc. | Chiral control |
| JPWO2015108046A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤 |
| US10149905B2 (en) | 2014-01-15 | 2018-12-11 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having antitumor effect and antitumor agent |
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| PL3237618T3 (pl) * | 2014-12-24 | 2019-09-30 | Uniqure Ip B.V. | Supresja genu huntingtyny indukowana rnai |
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| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
| US10457940B2 (en) * | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
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| EP3634953B1 (en) | 2017-06-05 | 2024-01-03 | PTC Therapeutics, Inc. | Compounds for treating huntington's disease |
| EP3642341A4 (en) | 2017-06-23 | 2021-06-16 | University Of Massachusetts | TWO-DAY SELF-RELEASING SIRNA AND RELATED PROCEDURES |
| US11395822B2 (en) | 2017-06-28 | 2022-07-26 | Ptc Therapeutics, Inc. | Methods for treating Huntington's disease |
| CA3067591A1 (en) | 2017-06-28 | 2019-01-03 | Ptc Therapeutics, Inc. | Methods for treating huntington's disease |
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| EP3697908A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| EP4124658A3 (en) | 2017-10-16 | 2023-04-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| CN108384803A (zh) * | 2017-12-19 | 2018-08-10 | 上海百力格生物技术有限公司 | 一种用于miRNA干扰载体构建筛选特异对照的质粒及其构建方法 |
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| US20210222196A1 (en) * | 2018-05-15 | 2021-07-22 | University Of Massachusetts | Modified aav constructs and uses thereof |
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| US8258286B2 (en) * | 2007-04-26 | 2012-09-04 | University Of Iowa Research Foundation | Reduction of off-target RNA interference toxicity |
| US20090036395A1 (en) * | 2007-04-26 | 2009-02-05 | Davidson Beverly L | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
| BRPI0814459B1 (pt) | 2007-07-26 | 2023-01-24 | Uniqure Ip B.V | Método para produzir um vírion parvoviral recombinante em uma célula de inseto, e, construção de ácido nucleico |
| WO2009104964A1 (en) | 2008-02-19 | 2009-08-27 | Amsterdam Molecular Therapeutics B.V. | Optimisation of expression of parvoviral rep and cap proteins in insect cells |
| EP2411505A4 (en) * | 2009-03-26 | 2013-01-30 | Univ California | MESENCHYMAL STEM CELLS PRODUCING INHIBITORY RNA WHICH CAN BE USED TO ACT IN THE COURSE OF A DISEASE |
| CA2759801C (en) * | 2009-05-02 | 2019-04-02 | Marco A. Passini | Gene therapy for neurodegenerative disorders |
| WO2011122950A1 (en) | 2010-04-01 | 2011-10-06 | Amsterdam Molecular Therapeutics (Amt) Ip B.V. | Monomeric duplex aav vectors |
| EP3502254A1 (en) * | 2010-04-23 | 2019-06-26 | Cold Spring Harbor Laboratory | Novel structurally designed shrnas |
| JP5941842B2 (ja) * | 2010-06-18 | 2016-06-29 | Ｌｓｉｐファンド運営合同会社 | 優性アレル発現抑制剤 |
| AU2012304993B2 (en) | 2011-09-08 | 2017-09-14 | 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 |
| PL3237618T3 (pl) * | 2014-12-24 | 2019-09-30 | Uniqure Ip B.V. | Supresja genu huntingtyny indukowana rnai |
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