JP2005506340A - 血液脳関門をわたる過小グリコシル化タンパク質の標的化のための方法および組成物 - Google Patents

血液脳関門をわたる過小グリコシル化タンパク質の標的化のための方法および組成物 Download PDF

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JP2005506340A
JP2005506340A JP2003535542A JP2003535542A JP2005506340A JP 2005506340 A JP2005506340 A JP 2005506340A JP 2003535542 A JP2003535542 A JP 2003535542A JP 2003535542 A JP2003535542 A JP 2003535542A JP 2005506340 A JP2005506340 A JP 2005506340A
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igf
therapeutic
protein
fusion protein
targeting
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JP2005506340A5 (https=
Inventor
ジョナサン エイチ． レボウィッツ，
ウィリアム エス． スライ，
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シムバイオンティクス インコーポレイテッド
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Priority claimed from US10/136,639 external-priority patent/US20030072761A1/en
Priority claimed from US10/136,841 external-priority patent/US7396811B2/en
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Publication of JP2005506340A publication Critical patent/JP2005506340A/ja
Publication of JP2005506340A5 publication Critical patent/JP2005506340A5/ja
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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
    • C12N15/62—DNA sequences coding for fusion proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/30—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • 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
    • A61K47/642—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 the peptide or protein in the drug conjugate being a cytokine, e.g. IL2, chemokine, growth factors or interferons being the inactive part of the conjugate
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • 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
    • A61K47/6425—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 the peptide or protein in the drug conjugate being a receptor, e.g. CD4, a cell surface antigen, i.e. not a peptide ligand targeting the antigen, or a cell surface determinant, i.e. a part of the surface of a cell
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/20—Fusion polypeptide containing a tag with affinity for a non-protein ligand
    • C07K2319/21—Fusion polypeptide containing a tag with affinity for a non-protein ligand containing a His-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/61—Fusion polypeptide containing an enzyme fusion for detection (lacZ, luciferase)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/74—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/74—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor
    • C07K2319/75—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor containing a fusion for activation of a cell surface receptor, e.g. thrombopoeitin, NPY and other peptide hormones

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Urology & Nephrology (AREA)
  • Endocrinology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Cardiology (AREA)
  • Neurosurgery (AREA)
  • Physics & Mathematics (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cell Biology (AREA)
  • Neurology (AREA)
  • Vascular Medicine (AREA)
JP2003535542A 2001-10-16 2002-10-16 血液脳関門をわたる過小グリコシル化タンパク質の標的化のための方法および組成物 Pending JP2005506340A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US32965001P 2001-10-16 2001-10-16
US10/136,639 US20030072761A1 (en) 2001-10-16 2002-04-30 Methods and compositions for targeting proteins across the blood brain barrier
US10/136,841 US7396811B2 (en) 2001-04-30 2002-04-30 Subcellular targeting of therapeutic proteins
US38445202P 2002-05-29 2002-05-29
US38601902P 2002-06-05 2002-06-05
US40881602P 2002-09-06 2002-09-06
PCT/US2002/032968 WO2003032727A1 (en) 2001-10-16 2002-10-16 Methods and compositions for targeting underglycosylated proteins across the blood brain barrier

Related Child Applications (1)

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JP2009143436A Division JP2009203241A (ja) 2001-10-16 2009-06-16 血液脳関門をわたる過小グリコシル化タンパク質の標的化のための方法および組成物

Publications (2)

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JP2005506340A true JP2005506340A (ja) 2005-03-03
JP2005506340A5 JP2005506340A5 (https=) 2006-01-12

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JP2003535542A Pending JP2005506340A (ja) 2001-10-16 2002-10-16 血液脳関門をわたる過小グリコシル化タンパク質の標的化のための方法および組成物
JP2009143436A Pending JP2009203241A (ja) 2001-10-16 2009-06-16 血液脳関門をわたる過小グリコシル化タンパク質の標的化のための方法および組成物

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EP (1) EP1446007A4 (https=)
JP (2) JP2005506340A (https=)
AU (3) AU2002347910A1 (https=)
CA (1) CA2463473A1 (https=)
IL (1) IL161352A0 (https=)
WO (2) WO2003032913A2 (https=)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013542913A (ja) * 2010-06-25 2013-11-28 シャイアー ヒューマン ジェネティック セラピーズ， インコーポレイテッド サンフィリポ症候群ｂ型の処置
JP2015505824A (ja) * 2011-12-01 2015-02-26 アンジオケム インコーポレーテッド 標的化リソソーム酵素化合物
JP2016094451A (ja) * 2010-06-25 2016-05-26 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド サンフィリポ症候群ｂ型の処置

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US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
ATE384736T1 (de) 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
WO2003032913A2 (en) * 2001-10-16 2003-04-24 Symbiontics Inc. Methods and compositions for targeting proteins across the blood brain barrier
CA2487815A1 (en) * 2002-05-29 2003-12-11 Biomarin Pharmaceutical Inc. Targeted therapeutic proteins
CN1922313B (zh) * 2004-02-10 2011-10-26 生物马林医药公司 酸性α-糖苷酶及其片段
AU2008282496B2 (en) 2007-07-27 2013-04-04 Armagen Technologies, Inc. Methods and compositions for increasing alpha-iduronidase activity in the CNS
KR101247418B1 (ko) * 2007-12-21 2013-03-25 에프. 호프만-라 로슈 아게 항체 제형
HRP20170698T1 (hr) 2008-05-07 2017-09-22 Biomarin Pharmaceutical Inc. Lizosomski usmjereni peptidi i njihove uporabe
EP2475376B1 (en) 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
JP2013507131A (ja) 2009-10-09 2013-03-04 アーメイゲン・テクノロジーズ・インコーポレイテッド Ｃｎｓにおけるイズロン酸２−スルファターゼ活性を増加させるための方法および組成物
SMT201600385T1 (it) 2010-06-25 2017-03-08 Shire Human Genetic Therapies Trasporto di agenti terapeutici all’snc
CN103179980B (zh) 2010-06-25 2016-09-28 夏尔人类遗传性治疗公司 艾杜糖醛酸-2-硫酸酯酶的cns递送的方法和组合物
NZ605873A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of arylsulfatase a
NZ605874A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of heparan n-sulfatase
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
EP2885318A4 (en) 2012-08-14 2016-03-30 Angiochem Inc PEPTIDE DENDRIMER CONJUGATES AND ITS USES
HUE039334T2 (hu) 2012-11-27 2018-12-28 Biomarin Pharm Inc Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai
JP6541581B2 (ja) * 2013-03-15 2019-07-10 グラクソスミスクライン、インテレクチュアル、プロパティー、（ナンバー２）、リミテッドＧｌａｘｏｓｍｉｔｈｋｌｉｎｅ Ｉｎｔｅｌｌｅｃｔｕａｌ Ｐｒｏｐｅｒｔｙ （Ｎｏ．２） Ｌｉｍｉｔｅｄ 低濃度抗体製剤
HUE039691T2 (hu) 2014-04-01 2019-01-28 Swedish Orphan Biovitrum Ab Publ Módosított szulfamidáz és elõállítása
DK3307326T3 (da) 2015-06-15 2020-10-19 Angiochem Inc Fremgangsmåder til behandling af leptomeningeal karcinomatose
EP4275751A3 (en) 2016-02-24 2023-12-27 BioMarin Pharmaceutical Inc. Improved naglu fusion protein formulation
KR20210005154A (ko) * 2018-04-30 2021-01-13 아미쿠스 세라퓨틱스, 인코포레이티드 유전자 요법 작제물 및 사용 방법
AR116624A1 (es) 2018-10-10 2021-05-26 Amicus Therapeutics Inc Composiciones de polipéptidos estabilizados por enlaces disulfuro y métodos de uso
JP7458404B2 (ja) 2018-12-20 2024-03-29 アーマジェン・インコーポレイテッド イズロン酸－２－スルファターゼ免疫グロブリン融合タンパク質の精製
EP4291246A4 (en) * 2021-02-11 2024-12-11 Denali Therapeutics Inc. ANTI-TRANSFERRIN RECEPTOR FUSION PROTEINS AND METHODS OF USE THEREOF

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US4801575A (en) * 1986-07-30 1989-01-31 The Regents Of The University Of California Chimeric peptides for neuropeptide delivery through the blood-brain barrier
US5672683A (en) * 1989-09-07 1997-09-30 Alkermes, Inc. Transferrin neuropharmaceutical agent fusion protein
CA2025907A1 (en) * 1989-09-21 1991-03-22 Franklin D. Collins Method of transporting compositions across the blood brain barrier
US6472140B1 (en) * 1997-09-05 2002-10-29 The General Hospital Corporation α-2- macroglobulin therapies and drug screening methods for Alzheimer's disease.
ATE384736T1 (de) * 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
WO2003032913A2 (en) * 2001-10-16 2003-04-24 Symbiontics Inc. Methods and compositions for targeting proteins across the blood brain barrier
CA2487815A1 (en) * 2002-05-29 2003-12-11 Biomarin Pharmaceutical Inc. Targeted therapeutic proteins

Cited By (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013542913A (ja) * 2010-06-25 2013-11-28 シャイアー ヒューマン ジェネティック セラピーズ， インコーポレイテッド サンフィリポ症候群ｂ型の処置
JP2016094451A (ja) * 2010-06-25 2016-05-26 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド サンフィリポ症候群ｂ型の処置
US9814764B2 (en) 2010-06-25 2017-11-14 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b by intrathecal administration of alpha-n-acetylglucosaminidase
US11065307B2 (en) 2010-06-25 2021-07-20 Shire Human Genetic Therapies, Inc. Therapeutic fusion protein comprising an alpha-n-acetylglucosaminidase and a lysosomal targeting moiety
US11065308B2 (en) 2010-06-25 2021-07-20 Shire Human Genetic Therapies, Inc. Methods and compositions for CNS delivery of heparan n-sulfatase
US11260112B2 (en) 2010-06-25 2022-03-01 Shire Human Genetic Therapies, Inc. Methods and compositions for CNS delivery of iduronate-2-sulfatase
US12121569B2 (en) 2010-06-25 2024-10-22 Takeda Pharmaceutical Company Limited Methods and compositions for CNS delivery of iduronate-2-sulfatase
US12161702B2 (en) 2010-06-25 2024-12-10 Takeda Pharmaceutical Company Limited Methods and compositions for CNS delivery of heparan N-sulfatase
US12168041B2 (en) 2010-06-25 2024-12-17 Takeda Pharmaceutical Company Limited Methods and compositions for CNS delivery of arylsulfatase A
US12409210B2 (en) 2010-06-25 2025-09-09 Takeda Pharmaceutical Company Limited CNS delivery of therapeutic agents
JP2015505824A (ja) * 2011-12-01 2015-02-26 アンジオケム インコーポレーテッド 標的化リソソーム酵素化合物

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WO2003032913A3 (en) 2004-08-12
WO2003032913A2 (en) 2003-04-24
JP2009203241A (ja) 2009-09-10
AU2010214643A1 (en) 2010-09-16
WO2003032913A9 (en) 2004-04-29
AU2002362930A2 (en) 2003-04-28
WO2003032727A1 (en) 2003-04-24
IL161352A0 (en) 2004-09-27
AU2002347910A1 (en) 2003-04-28
EP1446007A1 (en) 2004-08-18
CA2463473A1 (en) 2003-04-24
EP1446007A4 (en) 2005-12-07

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