JP2016522674A - 遺伝子発現を調節するための組成物及び方法 - Google Patents

遺伝子発現を調節するための組成物及び方法 Download PDF

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JP2016522674A
JP2016522674A JP2015512857A JP2015512857A JP2016522674A JP 2016522674 A JP2016522674 A JP 2016522674A JP 2015512857 A JP2015512857 A JP 2015512857A JP 2015512857 A JP2015512857 A JP 2015512857A JP 2016522674 A JP2016522674 A JP 2016522674A
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oligonucleotide
nucleotide
stranded oligonucleotide
nucleotides
sequence
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JP2016522674A5 (https=
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アーサー エム． クリーグ，
アーサー エム． クリーグ，
ロメシュ スブラマニアン，
ロメシュ スブラマニアン，
ジェイムズ マクスウィゲン，
ジェイムズ マクスウィゲン，
ジェニー ティー． リー，
ジェニー ティー． リー，
Original Assignee
ラナ セラピューティクス インコーポレイテッド
ラナ セラピューティクス インコーポレイテッド
ザ ジェネラル ホスピタル コーポレイション
ザ ジェネラル ホスピタル コーポレイション
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    • 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
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    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3517—Marker; Tag

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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2015512857A 2012-05-16 2013-05-16 遺伝子発現を調節するための組成物及び方法 Pending JP2016522674A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261648077P 2012-05-16 2012-05-16
US61/648,077 2012-05-16
PCT/US2013/041437 WO2013173637A1 (en) 2012-05-16 2013-05-16 Compositions and methods for modulating gene expression

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JP2016522674A true JP2016522674A (ja) 2016-08-04
JP2016522674A5 JP2016522674A5 (https=) 2016-09-15

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US (1) US20150141320A1 (https=)
EP (1) EP2850184A4 (https=)
JP (1) JP2016522674A (https=)
WO (1) WO2013173637A1 (https=)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021514182A (ja) * 2018-02-09 2021-06-10 ジェネンテック， インコーポレイテッド Ｔｍｅｍ１０６ｂ発現を調節するためのオリゴヌクレオチド
JP2023503202A (ja) * 2020-02-12 2023-01-26 アソキュラ・ファーマシューティカルズ・スージョウ・カンパニー，リミテッド アンチセンスオリゴヌクレオチドおよびペンドレッド症候群を処置するためのそれらの使用
JP2023523622A (ja) * 2020-04-29 2023-06-06 サリオジェン セラピューティクス インコーポレイテッド 遺伝性黄斑変性症の治療のための組成物及び方法

Families Citing this family (90)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2595663A4 (en) 2010-07-19 2014-03-05 Isis Pharmaceuticals Inc MODULATION OF DYSTROPHIA MYOTONICA PROTEIN KINASE (DMPK) EXPRESSION
CA2817256A1 (en) 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EA201492122A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии utrn
AU2013262699A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating ATP2A2 expression
SG11201407483YA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
BR112014028631A2 (pt) 2012-05-16 2017-10-17 Rana Therapeutics Inc composições e métodos para modulação da expressão da família de genes da hemoglobina
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
JP2016523103A (ja) * 2013-06-26 2016-08-08 エランコ・ユーエス・インコーポレイテッドＥｌａｎｃｏ ＵＳ Ｉｎｃ． イヌ糸状虫病の病因であるディロフィラリア・イミティス（Ｄｉｒｏｆｉｌａｒｉａ ｉｍｍｉｔｉｓ）において大環状ラクトン耐性を予測するためのマーカー
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
US11236338B2 (en) * 2013-09-05 2022-02-01 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
WO2015051283A1 (en) 2013-10-04 2015-04-09 Rana Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
EP3119888B1 (en) 2014-03-19 2021-07-28 Ionis Pharmaceuticals, Inc. Compositions for modulating ataxin 2 expression
HUE050704T2 (hu) 2014-04-01 2020-12-28 Biogen Ma Inc Összetételek a SOD-1 expressziójának modulálására
WO2016061487A1 (en) * 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
WO2016070060A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
US10793855B2 (en) * 2015-01-06 2020-10-06 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
AU2016222546B2 (en) 2015-02-26 2020-01-23 Ionis Pharmaceuticals, Inc. Allele specific modulators of P23H rhodopsin
GB201504124D0 (en) 2015-03-11 2015-04-22 Proqr Therapeutics B V Oligonucleotides
WO2016149455A2 (en) 2015-03-17 2016-09-22 The General Hospital Corporation The rna interactome of polycomb repressive complex 1 (prc1)
US10407678B2 (en) 2015-04-16 2019-09-10 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
US10801024B2 (en) * 2015-05-20 2020-10-13 Indiana University Research And Technology Corporation Inhibition of lncRNA HOTAIR and related materials and methods
EP3124619B1 (en) * 2015-07-31 2019-03-06 Menicon Co., Ltd Reagents, method and kit for across and within dog breed glaucoma diagnosis
JP2018538287A (ja) * 2015-12-14 2018-12-27 コールド スプリング ハーバー ラボラトリー 多発性嚢胞腎の処置のためのアンチセンスオリゴマー
AU2016381174A1 (en) 2015-12-31 2018-05-31 Ionis Pharmaceuticals, Inc. Methods for reducing Ataxin-2 expression
AU2017213404A1 (en) 2016-01-29 2018-09-20 Kyowa Kirin Co., Ltd. Nucleic acid conjugate
WO2017134252A1 (en) * 2016-02-05 2017-08-10 INSERM (Institut National de la Santé et de la Recherche Médicale) Antisense oligonucleotides effective to reduce the expression of menin in cancer cells of a subject
WO2017184529A1 (en) 2016-04-18 2017-10-26 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
JP2019514372A (ja) * 2016-04-20 2019-06-06 ジェービーエス サイエンス インコーポレイテッド Ｃｔｎｎｂ１およびｈｔｅｒｔにおける突然変異を検出するためのキットおよび方法、ならびにｈｃｃ検出および疾患管理におけるそれらの使用
AU2017286811A1 (en) 2016-06-17 2018-11-22 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
TWI746590B (zh) 2016-06-30 2021-11-21 日商協和麒麟股份有限公司 核酸複合體
MX2018015722A (es) 2016-07-01 2019-05-27 Hoffmann La Roche Oligonucleotidos antisentido para modular expresion del requisito de alta temperatura a 1 (htra1).
EP3481432A4 (en) * 2016-07-05 2020-05-06 The U.S.A. as represented by the Secretary, Department of Health and Human Services Diagnosing col6-related disorders and methods for treating same
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
US11118182B2 (en) 2016-11-17 2021-09-14 Ramot At Tel-Aviv University Ltd. Modulators of human KAI1 metastasis suppressor gene, methods and uses thereof
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
US11352625B2 (en) 2017-01-12 2022-06-07 Duke University Compositions and methods for disrupting the molecular mechanisms associated with mitochondrial dysfunction and neurodegenerative disease
JP7141621B2 (ja) * 2017-03-31 2022-09-26 学校法人 愛知医科大学 コンドロイチン硫酸生合成を阻害するアンチセンス核酸
GB201706009D0 (en) * 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
US20190055564A1 (en) * 2017-06-01 2019-02-21 F. Hoffmann-La Roche Ag Antisense oligonucleotides for modulating htra1 expression
US20200208152A1 (en) 2017-09-08 2020-07-02 Mina Therapeutics Limited Stabilized sarna compositions and methods of use
WO2019048632A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATORY RNA (PARNA) OF HNF4A AND METHODS OF USE
WO2019094315A1 (en) * 2017-11-08 2019-05-16 Aptamer Diagnostic, Inc. D-dimer-specific aptamers and methods of use in diagnostics, therapeutic and theranostic purposes
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
KR102723989B1 (ko) * 2017-12-01 2024-11-01 더 텍사스 에이 & 엠 유니버시티 시스템 엔젤만 증후군 안티센스 치료
MX2020008581A (es) * 2018-02-21 2020-09-21 Bristol Myers Squibb Co Oligonucleotidos antisentido de la proteina cinasa del tipo ii delta dependiente del calcio/calmodulina (camk2d) y sus usos.
TWI840345B (zh) 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
US11732260B2 (en) 2018-03-02 2023-08-22 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-β precursor protein
SG11202008660TA (en) 2018-04-11 2020-10-29 Ionis Pharmaceuticals Inc Modulators of ezh2 expression
EP4663758A3 (en) 2018-05-09 2026-04-29 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
TWI851574B (zh) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原（ＡＧＴ）ｉＲＮＡ組成物及其使用方法
WO2020007700A1 (en) * 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting spi1
WO2020007772A1 (en) * 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting gbp-1
WO2020011653A1 (en) * 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting kynu
WO2020015959A1 (en) * 2018-07-19 2020-01-23 Stichting Katholieke Universiteit Antisense oligonucleotides rescue aberrant splicing of abca4.
AR115847A1 (es) 2018-07-25 2021-03-03 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de la atxn2
JP7529286B2 (ja) * 2018-10-26 2024-08-06 フリーイェ・ユニヴェルシテイト・ブリュッセル 肝特異的核酸調節エレメント並びにその方法及び使用
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
BR112021013369A2 (pt) 2019-01-31 2021-09-21 Ionis Pharmaceuticals, Inc. Moduladores de expressão de yap1
CN113811311A (zh) * 2019-03-15 2021-12-17 马萨诸塞大学 用于组织特异性apoe调节的寡核苷酸
PT3947684T (pt) 2019-03-29 2025-05-19 Ionis Pharmaceuticals Inc Compostos e métodos para modular ube3a‑ats
US11739324B2 (en) 2019-06-25 2023-08-29 Stichting Katholieke Universiteit Antisense oligonucleotides rescue aberrant splicing of ABCA4
US20220281911A1 (en) 2019-08-19 2022-09-08 Mina Therapeutics Limited Oligonucleotide conjugate compositions and methods of use
CN115279902A (zh) * 2019-08-30 2022-11-01 贝勒医学院 用于调控基因表达的系统
EP3822370A1 (en) * 2019-11-15 2021-05-19 Curiosity Diagnostics Sp. z o.o. Method of determining the presence of a hyper-virulent clostridioides difficile strain of the b1/nap1/027 group in a sample
WO2021126997A1 (en) * 2019-12-18 2021-06-24 Petdx, Inc. Methods and compositions for cancer detection, characterization or management in companion animals
KR20220133925A (ko) * 2020-01-31 2022-10-05 가부시키가이샤산와카가쿠켄큐쇼 Atn1의 안티센스 올리고뉴클레오티드
EP4116419A1 (en) * 2020-03-04 2023-01-11 Nissan Chemical Corporation Antisense oligonucleotide of calm2
EP4172338A4 (en) 2020-06-29 2025-06-11 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING PLP1
KR20230095056A (ko) * 2020-07-22 2023-06-28 더 플로레이 인스티튜트 오브 뉴로사이언스 앤드 멘탈 헬스 Scn2a에서 기능-상실 돌연변이와 연관된 장애를 치료하기 위한 조성물 및 방법
CA3189922A1 (en) 2020-07-28 2022-02-03 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
WO2022032017A2 (en) * 2020-08-07 2022-02-10 The General Hospital Corporation Human xist antisense oligonucleotides for x reactivation therapy
EP4015040A1 (en) * 2020-12-16 2022-06-22 Fundación para la Formación e Investigación Sanitarias de la Región de Murcia Method for treating blood diseases
WO2022133278A2 (en) 2020-12-18 2022-06-23 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating factor xii
WO2022232687A1 (en) * 2021-04-30 2022-11-03 Greenlight Biosciences, Inc. Messenger rna therapeutics and compositions
AU2022293556A1 (en) 2021-06-18 2024-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ifnar1 expression
WO2023004049A1 (en) * 2021-07-21 2023-01-26 AcuraStem, Inc. Unc13a antisense oligonucleotides
WO2023034870A2 (en) 2021-09-01 2023-03-09 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing dmpk expression
WO2023111337A1 (en) * 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Antisense oligonucleotide
WO2023139360A1 (en) * 2022-01-19 2023-07-27 Autolus Limited Nucleic acid construct
WO2024064608A2 (en) * 2022-09-20 2024-03-28 Intergalactic Therapeutics, Inc. Best1 vectors and uses thereof
GB202312098D0 (en) * 2023-08-08 2023-09-20 Ucl Business Ltd Therapeutic molecules
AU2024339330A1 (en) * 2023-09-13 2026-04-02 Haya Therapeutics Sa Compositions and methods for modulating wisp2 super-enhancer-associated rna
WO2025072672A2 (en) * 2023-09-27 2025-04-03 Judo Bio, Inc. Slc6a19-targeting modulatory nucleic acid agents
GB202401412D0 (en) * 2024-02-02 2024-03-20 Harness Therapeutics Ltd Functional nucleic acid
WO2026033502A1 (en) * 2024-08-09 2026-02-12 Associação Biopolis Kit, method, and uses thereof
WO2026074434A1 (en) * 2024-10-01 2026-04-09 Fondazione Istituto Italiano Di Tecnologia Rad51/brca2 complex aptamers and uses thereof

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH04320700A (ja) * 1991-04-17 1992-11-11 Nippon Shoji Kk アポリポプロテインｅ遺伝子タイプの検査方法及びその検査に好適なプライマー及びプローブ
WO2008103763A2 (en) * 2007-02-20 2008-08-28 Sequenom, Inc. Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation
JP2010516256A (ja) * 2007-01-19 2010-05-20 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン Ａｄｒｂ２癌マーカー
US20120004278A1 (en) * 2010-06-18 2012-01-05 The Board Of Trustees Of The Leland Stanford Junior University Linc rnas in cancer diagnosis and treatment

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008025069A1 (en) * 2006-08-28 2008-03-06 The Walter And Eliza Hall Institute Of Medical Research Methods of modulating cellular activity and compositions therefor
EP2331543A4 (en) * 2008-09-26 2013-06-19 Agency Science Tech & Res 3-deazaneplanocin DERIVATIVES

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH04320700A (ja) * 1991-04-17 1992-11-11 Nippon Shoji Kk アポリポプロテインｅ遺伝子タイプの検査方法及びその検査に好適なプライマー及びプローブ
JP2010516256A (ja) * 2007-01-19 2010-05-20 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン Ａｄｒｂ２癌マーカー
WO2008103763A2 (en) * 2007-02-20 2008-08-28 Sequenom, Inc. Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation
US20120004278A1 (en) * 2010-06-18 2012-01-05 The Board Of Trustees Of The Leland Stanford Junior University Linc rnas in cancer diagnosis and treatment

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
KANHERE A. ET AL., MOLECULAR CELL, vol. 38(2010), JPN6017005739, pages 675 - 688, ISSN: 0003506431 *
PRENSNER J.R. ET AL., NAT. BIOTECHNOL., vol. 29(8)(2011), JPN6017005745, pages 742 - 749, ISSN: 0003506433 *
SARMA K. ET AL., PROC. NATL. ACAD. USA, vol. 107(51)(2010), JPN6017005742, pages 22196 - 22201, ISSN: 0003506430 *
ZHAO J. ET AL., SCIENCE, vol. 322(5902)(2008), JPN6017005741, pages 750 - 756, ISSN: 0003506429 *
神武洋二郎 他: "長鎖ｎｃＲＮＡによるクロマチン修飾因子のリクルートメント機構", 実験医学, vol. Vol.29 No.11(2011), JPN7017000587, pages 1716 - 1721, ISSN: 0003506432 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021514182A (ja) * 2018-02-09 2021-06-10 ジェネンテック， インコーポレイテッド Ｔｍｅｍ１０６ｂ発現を調節するためのオリゴヌクレオチド
JP7281474B2 (ja) 2018-02-09 2023-05-25 ジェネンテック， インコーポレイテッド Ｔｍｅｍ１０６ｂ発現を調節するためのオリゴヌクレオチド
JP2023503202A (ja) * 2020-02-12 2023-01-26 アソキュラ・ファーマシューティカルズ・スージョウ・カンパニー，リミテッド アンチセンスオリゴヌクレオチドおよびペンドレッド症候群を処置するためのそれらの使用
JP7321614B2 (ja) 2020-02-12 2023-08-07 アソキュラ・ファーマシューティカルズ・スージョウ・カンパニー，リミテッド アンチセンスオリゴヌクレオチドおよびペンドレッド症候群を処置するためのそれらの使用
JP2023523622A (ja) * 2020-04-29 2023-06-06 サリオジェン セラピューティクス インコーポレイテッド 遺伝性黄斑変性症の治療のための組成物及び方法

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