TR201908969T4 - Rnaı indüklü huntıngtın geni supresyonu. - Google Patents
Rnaı indüklü huntıngtın geni supresyonu. Download PDFInfo
- Publication number
- TR201908969T4 TR201908969T4 TR2019/08969T TR201908969T TR201908969T4 TR 201908969 T4 TR201908969 T4 TR 201908969T4 TR 2019/08969 T TR2019/08969 T TR 2019/08969T TR 201908969 T TR201908969 T TR 201908969T TR 201908969 T4 TR201908969 T4 TR 201908969T4
- Authority
- TR
- Turkey
- Prior art keywords
- rna
- gene suppression
- sequence
- huntingtin gene
- rna sequence
- Prior art date
Links
- 108091032973 (ribonucleotides)n+m Proteins 0.000 title abstract 4
- 108090000623 proteins and genes Proteins 0.000 title 1
- 230000001629 suppression Effects 0.000 title 1
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 4
- 102000040650 (ribonucleotides)n+m Human genes 0.000 abstract 3
- 230000000295 complement effect Effects 0.000 abstract 2
- 108091030071 RNAI Proteins 0.000 abstract 1
- 238000010171 animal model Methods 0.000 abstract 1
- 230000030833 cell death Effects 0.000 abstract 1
- 230000009368 gene silencing by RNA Effects 0.000 abstract 1
- 230000001939 inductive effect Effects 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 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
- A61K31/713—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
-
- 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
Abstract
Mevcut buluş; bir birinci RNA sekansı ve bir ikinci RNA sekansını içeren bir çift dizgili RNA sağlamaktadır, burada birinci ve ikinci RNA sekansı büyük ölçüde tamamlayıcıdır, burada birinci RNA sekansı, en az 19 nükleotitlik bir sekans uzunluğuna sahiptir ve büyük ölçüde SEKANS KİMLİK NUMARASI: 1'e tamamlayıcıdır. Söz konusu çift dizgili RNA, Huntingtin ekzon 1 sekanslarına karşı RNAi'nin indüklenmesine kullanılmaya yöneliktir. Buluşa ait çift dizgili RNA, bir hayvan modelinde nöronol hücre ölümünü ve huntingtin agregalarının azaltabilmektedir.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP14200308 | 2014-12-24 |
Publications (1)
Publication Number | Publication Date |
---|---|
TR201908969T4 true TR201908969T4 (tr) | 2019-07-22 |
Family
ID=52282540
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
TR2019/08969T TR201908969T4 (tr) | 2014-12-24 | 2015-12-23 | Rnaı indüklü huntıngtın geni supresyonu. |
Country Status (25)
Country | Link |
---|---|
US (4) | US10174321B2 (tr) |
EP (2) | EP3237618B1 (tr) |
JP (2) | JP6839094B2 (tr) |
KR (1) | KR102636633B1 (tr) |
CN (1) | CN108064292B (tr) |
AU (1) | AU2015370903B2 (tr) |
BR (1) | BR112017013573A2 (tr) |
CA (1) | CA2971920A1 (tr) |
CO (1) | CO2017007335A2 (tr) |
CY (1) | CY1121733T1 (tr) |
DK (1) | DK3237618T3 (tr) |
EA (1) | EA037696B1 (tr) |
ES (1) | ES2732023T3 (tr) |
HK (1) | HK1246344B (tr) |
HR (1) | HRP20190992T1 (tr) |
HU (1) | HUE043842T2 (tr) |
IL (1) | IL252990B (tr) |
LT (1) | LT3237618T (tr) |
MX (1) | MX2017008500A (tr) |
PL (1) | PL3237618T3 (tr) |
PT (1) | PT3237618T (tr) |
RS (1) | RS58862B1 (tr) |
SI (1) | SI3237618T1 (tr) |
TR (1) | TR201908969T4 (tr) |
WO (1) | WO2016102664A1 (tr) |
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WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
EP2734208B1 (en) | 2011-07-19 | 2017-03-01 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
WO2014010718A1 (ja) | 2012-07-13 | 2014-01-16 | 株式会社新日本科学 | キラル核酸アジュバント |
KR102213609B1 (ko) | 2012-07-13 | 2021-02-08 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
EP2872485B1 (en) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
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 |
AU2015207773B2 (en) | 2014-01-16 | 2021-06-17 | Wave Life Sciences Ltd. | Chiral design |
EP3218484A4 (en) | 2014-11-14 | 2018-05-30 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
SG11201703419UA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Modulatory polynucleotides |
PT3237618T (pt) * | 2014-12-24 | 2019-07-04 | Uniqure Ip Bv | Supressão do gene huntingtina induzida por arni |
JP2018506304A (ja) | 2015-02-10 | 2018-03-08 | ジェンザイム・コーポレーション | バリアントRNAi |
MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
LT3386511T (lt) | 2015-12-10 | 2021-08-25 | Ptc Therapeutics, Inc. | Hantingtono ligos gydymo būdai |
MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
WO2017201258A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
EP3458588A4 (en) | 2016-05-18 | 2020-01-15 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
US10457940B2 (en) * | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
WO2018204803A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
EP3634953B1 (en) | 2017-06-05 | 2024-01-03 | PTC Therapeutics, Inc. | Compounds for treating huntington's disease |
CN111182898B (zh) | 2017-06-28 | 2024-04-16 | Ptc医疗公司 | 用于治疗亨廷顿氏病的方法 |
EP3645121A4 (en) | 2017-06-28 | 2021-03-17 | PTC Therapeutics, Inc. | HUNTINGTON'S DISEASE TREATMENT METHODS |
CA3076191A1 (en) * | 2017-09-22 | 2019-03-28 | Genzyme Corporation | Variant rnai |
AU2018352236A1 (en) | 2017-10-16 | 2020-04-23 | The Curators Of The University Of Missouri | Treatment of amyotrophic lateral sclerosis (ALS) |
WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | 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 |
US20210009590A1 (en) | 2018-03-27 | 2021-01-14 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
US20210269825A1 (en) * | 2018-05-15 | 2021-09-02 | University Of Washington | Compositions and methods for reducing spliceopathy and treating rna dominance disorders |
EP3794125A4 (en) * | 2018-05-15 | 2022-07-13 | University of Massachusetts | MODIFIED AAV CONSTRUCTS AND USES THEREOF |
EP3814357B1 (en) | 2018-06-27 | 2024-05-01 | PTC Therapeutics, Inc. | Heterocyclic and heteroaryl compounds for treating huntington's disease |
JP2021529513A (ja) | 2018-07-02 | 2021-11-04 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | 筋萎縮性側索硬化症および脊髄に関連する障害の治療 |
EP3850098A1 (en) | 2018-09-12 | 2021-07-21 | uniQure IP B.V. | Rnai induced c9orf72 suppression for the treatment of als/ftd |
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WO2020104295A1 (en) | 2018-11-19 | 2020-05-28 | Uniqure Ip B.V. | Rnai induced reduction of ataxin-3 for the treatment of spinocerebellar ataxia type 3 |
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JP2023521347A (ja) * | 2020-04-07 | 2023-05-24 | ユニキュアー アイピー ビー.ブイ. | アンジオポエチン様3(angptl3)をサイレンシングするための遺伝子構築物及びその使用 |
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WO2022031591A2 (en) * | 2020-08-03 | 2022-02-10 | University Of Massachusetts | Oligonucleotides for htt-1a modulation |
TW202304446A (zh) | 2021-03-29 | 2023-02-01 | 瑞士商諾華公司 | 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途 |
JP2024511207A (ja) * | 2021-03-30 | 2024-03-12 | 南京大学 | ハンチントン病を治療するためのrna送達システム |
EP4359525A1 (en) | 2021-06-21 | 2024-05-01 | uniQure biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
WO2023147058A2 (en) * | 2022-01-27 | 2023-08-03 | Asklepios Biopharmaceutical, Inc. | Compositions for treating neurological disease |
WO2023198745A1 (en) | 2022-04-12 | 2023-10-19 | Uniqure Biopharma B.V. | Nucleic acid regulation of apoe |
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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 |
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 |
DK2744895T3 (en) | 2011-09-08 | 2016-01-04 | Uniqure Ip Bv | REMOVAL OF INFECTED VIRA 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 |
PT3237618T (pt) * | 2014-12-24 | 2019-07-04 | Uniqure Ip Bv | Supressão do gene huntingtina induzida por arni |
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2015
- 2015-12-23 PT PT15817385T patent/PT3237618T/pt unknown
- 2015-12-23 RS RS20190744A patent/RS58862B1/sr unknown
- 2015-12-23 US US15/538,964 patent/US10174321B2/en active Active
- 2015-12-23 AU AU2015370903A patent/AU2015370903B2/en active Active
- 2015-12-23 EA EA201791417A patent/EA037696B1/ru unknown
- 2015-12-23 HU HUE15817385A patent/HUE043842T2/hu unknown
- 2015-12-23 EP EP15817385.6A patent/EP3237618B1/en active Active
- 2015-12-23 MX MX2017008500A patent/MX2017008500A/es unknown
- 2015-12-23 CN CN201580071228.2A patent/CN108064292B/zh active Active
- 2015-12-23 TR TR2019/08969T patent/TR201908969T4/tr unknown
- 2015-12-23 KR KR1020177020454A patent/KR102636633B1/ko active IP Right Grant
- 2015-12-23 ES ES15817385T patent/ES2732023T3/es active Active
- 2015-12-23 BR BR112017013573A patent/BR112017013573A2/pt active Search and Examination
- 2015-12-23 SI SI201530791T patent/SI3237618T1/sl unknown
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- 2015-12-23 PL PL15817385T patent/PL3237618T3/pl unknown
- 2015-12-23 EP EP19167349.0A patent/EP3540063A1/en active Pending
- 2015-12-23 LT LTEP15817385.6T patent/LT3237618T/lt unknown
- 2015-12-23 DK DK15817385.6T patent/DK3237618T3/da active
- 2015-12-23 WO PCT/EP2015/081157 patent/WO2016102664A1/en active Application Filing
- 2015-12-23 JP JP2017552234A patent/JP6839094B2/ja active Active
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2017
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2018
- 2018-04-27 HK HK18105533.3A patent/HK1246344B/zh unknown
- 2018-11-16 US US16/193,908 patent/US10767180B2/en active Active
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- 2019-05-31 HR HRP20190992TT patent/HRP20190992T1/hr unknown
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- 2020-07-15 US US16/930,035 patent/US11371044B2/en active Active
- 2020-10-01 JP JP2020167128A patent/JP2021000136A/ja active Pending
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2022
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