PL3237618T3 - Supresja genu huntingtyny indukowana rnai - Google Patents
Supresja genu huntingtyny indukowana rnaiInfo
- Publication number
- PL3237618T3 PL3237618T3 PL15817385T PL15817385T PL3237618T3 PL 3237618 T3 PL3237618 T3 PL 3237618T3 PL 15817385 T PL15817385 T PL 15817385T PL 15817385 T PL15817385 T PL 15817385T PL 3237618 T3 PL3237618 T3 PL 3237618T3
- Authority
- PL
- Poland
- Prior art keywords
- gene suppression
- huntingtin gene
- rnai induced
- induced huntingtin
- rnai
- Prior art date
Links
Classifications
-
- 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
-
- 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
-
- 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
-
- 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.
-
- 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
-
- 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
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP14200308 | 2014-12-24 | ||
EP15817385.6A EP3237618B1 (en) | 2014-12-24 | 2015-12-23 | Rnai induced huntingtin gene suppression |
PCT/EP2015/081157 WO2016102664A1 (en) | 2014-12-24 | 2015-12-23 | Rnai induced huntingtin gene suppression |
Publications (1)
Publication Number | Publication Date |
---|---|
PL3237618T3 true PL3237618T3 (pl) | 2019-09-30 |
Family
ID=52282540
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PL15817385T PL3237618T3 (pl) | 2014-12-24 | 2015-12-23 | Supresja genu huntingtyny indukowana rnai |
Country Status (25)
Country | Link |
---|---|
US (4) | US10174321B2 (pl) |
EP (2) | EP3237618B1 (pl) |
JP (2) | JP6839094B2 (pl) |
KR (1) | KR102636633B1 (pl) |
CN (1) | CN108064292B (pl) |
AU (1) | AU2015370903B2 (pl) |
BR (1) | BR112017013573A2 (pl) |
CA (1) | CA2971920A1 (pl) |
CO (1) | CO2017007335A2 (pl) |
CY (1) | CY1121733T1 (pl) |
DK (1) | DK3237618T3 (pl) |
EA (1) | EA037696B1 (pl) |
ES (1) | ES2732023T3 (pl) |
HK (1) | HK1246344B (pl) |
HR (1) | HRP20190992T1 (pl) |
HU (1) | HUE043842T2 (pl) |
IL (1) | IL252990B (pl) |
LT (1) | LT3237618T (pl) |
MX (1) | MX2017008500A (pl) |
PL (1) | PL3237618T3 (pl) |
PT (1) | PT3237618T (pl) |
RS (1) | RS58862B1 (pl) |
SI (1) | SI3237618T1 (pl) |
TR (1) | TR201908969T4 (pl) |
WO (1) | WO2016102664A1 (pl) |
Families Citing this family (54)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
KR101881596B1 (ko) | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
EP2451461A4 (en) | 2009-07-06 | 2013-05-29 | Ontorii Inc | NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF |
JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 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 |
DK2872485T3 (da) | 2012-07-13 | 2021-03-08 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
CA2878945A1 (en) | 2012-07-13 | 2014-01-16 | Wave Life Sciences Pte. Ltd. | Chiral control |
SG11201500243WA (en) | 2012-07-13 | 2015-04-29 | Shin Nippon Biomedical Lab Ltd | Chiral nucleic acid adjuvant |
WO2015108046A1 (ja) | 2014-01-15 | 2015-07-23 | 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 |
EP3095461A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
EP3095459A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having antitumor effect and antitumor agent |
US10160969B2 (en) | 2014-01-16 | 2018-12-25 | Wave Life Sciences Ltd. | Chiral design |
CA3193811A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
WO2016077689A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
AU2015370903B2 (en) * | 2014-12-24 | 2021-06-17 | Uniqure Ip B.V. | RNAi induced huntingtin gene suppression |
EP3256588A2 (en) | 2015-02-10 | 2017-12-20 | Genzyme Corporation | VARIANT RNAi |
MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
KR102488323B1 (ko) | 2015-12-10 | 2023-01-12 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료 또는 개선을 위한 조성물 |
MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
IL297576B2 (en) | 2016-05-18 | 2024-02-01 | Voyager Therapeutics Inc | Preparations and methods for treating Huntington's disease |
MX2018014154A (es) | 2016-05-18 | 2019-05-06 | Voyager Therapeutics Inc | Polinucleotidos moduladores. |
US10457940B2 (en) * | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
MX2019013172A (es) | 2017-05-05 | 2020-09-07 | Voyager Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de huntington. |
MX2019014514A (es) | 2017-06-05 | 2020-07-20 | Ptc Therapeutics Inc | Compuestos para tratar la enfermedad de huntington. |
MX2019015578A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
MX2019015580A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
CN111356763B (zh) * | 2017-09-22 | 2024-03-12 | 建新公司 | 变体RNAi |
WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
JP2021502060A (ja) | 2017-10-16 | 2021-01-28 | 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(als)の治療 |
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 |
WO2019191092A1 (en) | 2018-03-27 | 2019-10-03 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
US20210222196A1 (en) * | 2018-05-15 | 2021-07-22 | University Of Massachusetts | Modified aav constructs and uses thereof |
BR112020023298A2 (pt) * | 2018-05-15 | 2021-03-09 | University Of Washington | composições e métodos para reduzir a spliceopatia e tratamento de distúrbios de dominância de rna |
KR20210042265A (ko) | 2018-06-27 | 2021-04-19 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료를 위한 헤테로사이클릭 및 헤테로아릴 화합물 |
CA3103963A1 (en) | 2018-07-02 | 2020-01-09 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
WO2020053258A1 (en) | 2018-09-12 | 2020-03-19 | Uniqure Ip B.V. | Rnai induced c9orf72 suppression for the treatment of als/ftd |
CA3121010A1 (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 |
AU2019382824A1 (en) | 2018-11-19 | 2021-06-03 | Uniqure Ip B.V. | Method and means to deliver miRNA to target cells |
CA3119721A1 (en) | 2018-11-19 | 2020-05-28 | Uniqure Ip B.V. | A companion diagnostic to monitor the effects of gene therapy |
EP3908658A1 (en) * | 2019-01-09 | 2021-11-17 | Universidade de Coimbra | Double stranded rna and uses thereof |
US20220251567A1 (en) | 2019-07-10 | 2022-08-11 | Inserm (Institut National De La Santè Et De La Recherche Médicale) | Methods for the treatment of epilepsy |
WO2021053018A1 (en) | 2019-09-16 | 2021-03-25 | Uniqure Ip B.V. | Targeting misspliced transcripts in genetic disorders |
AR120341A1 (es) * | 2019-11-01 | 2022-02-09 | Alnylam Pharmaceuticals Inc | COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO |
MX2022005254A (es) | 2019-11-01 | 2022-06-29 | Novartis Ag | El uso de un modulador de empalme para un tratamiento que retrasa la progresion de la enfermedad de huntington. |
EP4133074A1 (en) * | 2020-04-07 | 2023-02-15 | uniQure IP B.V. | Gene constructs for silencing angiopoietin-like 3 (angptl3) and uses thereof |
WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
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 |
EP4317434A1 (en) * | 2021-03-30 | 2024-02-07 | Nanjing University | Rna delivery system for treatment of huntington's disease |
WO2022268835A1 (en) | 2021-06-21 | 2022-12-29 | 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 |
WO2023198702A1 (en) | 2022-04-12 | 2023-10-19 | Uniqure Biopharma B.V. | Nucleic acid regulation of c9orf72 |
WO2023198745A1 (en) | 2022-04-12 | 2023-10-19 | Uniqure Biopharma B.V. | Nucleic acid regulation of apoe |
WO2023198662A1 (en) | 2022-04-12 | 2023-10-19 | Uniqure Biopharma B.V. | Novel systems for nucleic acid regulation |
WO2023198663A1 (en) | 2022-04-12 | 2023-10-19 | Uniqure Biopharma B.V. | Nucleic acid regulation of snca |
Family Cites Families (18)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU2004239114B2 (en) * | 2003-05-14 | 2008-03-13 | Japan Science And Technology Agency | Inhibition of the expression of huntingtin gene |
US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
WO2005105995A2 (en) | 2004-04-14 | 2005-11-10 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA) |
WO2007046703A2 (en) | 2005-10-20 | 2007-04-26 | Amsterdam Molecular Therapeutics B.V. | Improved aav vectors produced in insect cells |
CN101506369B (zh) | 2006-06-21 | 2014-02-12 | 尤尼克尔生物制药股份有限公司 | 具有经修饰的用于在昆虫细胞中产生aav的aav-rep78翻译起始密码子的载体 |
EP2046993A4 (en) | 2006-07-07 | 2010-11-17 | Univ Massachusetts | RNA INTERFERENCE COMPOSITIONS AND METHOD FOR TREATING MORBUS HUNTINGTON |
WO2008134646A2 (en) * | 2007-04-26 | 2008-11-06 | University Of Iowa Research Foundation | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
WO2008150897A2 (en) * | 2007-05-31 | 2008-12-11 | University Of Iowa Research Foundation | Reduction of off-target rna interference toxicity |
CN101868547B (zh) | 2007-07-26 | 2016-03-16 | 尤尼克尔Ip股份有限公司 | 包含具有差别密码子偏性的重复编码序列的杆状病毒载体 |
ES2929031T3 (es) | 2008-02-19 | 2022-11-24 | Uniqure Ip Bv | Optimización de la expresión de las proteínas rep y cap parvovirales en células de insecto |
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 |
KR101835490B1 (ko) * | 2009-05-02 | 2018-03-08 | 젠자임 코포레이션 | 신경변성 질환에 대한 유전자 요법 |
WO2011122950A1 (en) | 2010-04-01 | 2011-10-06 | Amsterdam Molecular Therapeutics (Amt) Ip B.V. | Monomeric duplex aav vectors |
LT2561078T (lt) * | 2010-04-23 | 2019-01-10 | Cold Spring Harbor Laboratory | Naujos struktūriškai sukonstruotos shrnr |
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 |
US9840694B2 (en) | 2011-09-08 | 2017-12-12 | 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 |
AU2015370903B2 (en) * | 2014-12-24 | 2021-06-17 | Uniqure Ip B.V. | RNAi induced huntingtin gene suppression |
-
2015
- 2015-12-23 AU AU2015370903A patent/AU2015370903B2/en active Active
- 2015-12-23 EP EP15817385.6A patent/EP3237618B1/en active Active
- 2015-12-23 KR KR1020177020454A patent/KR102636633B1/ko active IP Right Grant
- 2015-12-23 DK DK15817385.6T patent/DK3237618T3/da active
- 2015-12-23 BR BR112017013573A patent/BR112017013573A2/pt active Search and Examination
- 2015-12-23 PL PL15817385T patent/PL3237618T3/pl unknown
- 2015-12-23 EA EA201791417A patent/EA037696B1/ru unknown
- 2015-12-23 JP JP2017552234A patent/JP6839094B2/ja active Active
- 2015-12-23 LT LTEP15817385.6T patent/LT3237618T/lt unknown
- 2015-12-23 ES ES15817385T patent/ES2732023T3/es active Active
- 2015-12-23 CA CA2971920A patent/CA2971920A1/en active Pending
- 2015-12-23 WO PCT/EP2015/081157 patent/WO2016102664A1/en active Application Filing
- 2015-12-23 RS RS20190744A patent/RS58862B1/sr unknown
- 2015-12-23 PT PT15817385T patent/PT3237618T/pt unknown
- 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 US US15/538,964 patent/US10174321B2/en active Active
- 2015-12-23 HU HUE15817385A patent/HUE043842T2/hu unknown
- 2015-12-23 EP EP19167349.0A patent/EP3540063A1/en active Pending
- 2015-12-23 SI SI201530791T patent/SI3237618T1/sl unknown
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2017
- 2017-06-18 IL IL252990A patent/IL252990B/en unknown
- 2017-07-24 CO CONC2017/0007335A patent/CO2017007335A2/es unknown
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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
- 2019-05-31 HR HRP20190992TT patent/HRP20190992T1/hr unknown
- 2019-06-13 CY CY20191100616T patent/CY1121733T1/el unknown
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2020
- 2020-07-15 US US16/930,035 patent/US11371044B2/en active Active
- 2020-10-01 JP JP2020167128A patent/JP2021000136A/ja active Pending
-
2022
- 2022-05-24 US US17/751,934 patent/US20230119344A1/en active Pending
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