BR112015009746A2 - Agente que aumenta atividade de glicocerebrosidase em mamíferos - Google Patents
Agente que aumenta atividade de glicocerebrosidase em mamíferosInfo
- Publication number
- BR112015009746A2 BR112015009746A2 BR112015009746A BR112015009746A BR112015009746A2 BR 112015009746 A2 BR112015009746 A2 BR 112015009746A2 BR 112015009746 A BR112015009746 A BR 112015009746A BR 112015009746 A BR112015009746 A BR 112015009746A BR 112015009746 A2 BR112015009746 A2 BR 112015009746A2
- Authority
- BR
- Brazil
- Prior art keywords
- agent
- increases
- activity
- mammals
- mammal
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
- C07K16/40—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against enzymes
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- 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
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
- A61P25/16—Anti-Parkinson drugs
-
- 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
- A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
-
- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Psychiatry (AREA)
- Physics & Mathematics (AREA)
- Mycology (AREA)
- Dermatology (AREA)
- Psychology (AREA)
- Hospice & Palliative Care (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261722434P | 2012-11-05 | 2012-11-05 | |
| PCT/US2013/068242 WO2014071282A1 (en) | 2012-11-05 | 2013-11-04 | Compositions and methods for treating proteinopathies |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112015009746A2 true BR112015009746A2 (pt) | 2017-08-15 |
Family
ID=49578599
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112015009746A BR112015009746A2 (pt) | 2012-11-05 | 2013-11-04 | Agente que aumenta atividade de glicocerebrosidase em mamíferos |
Country Status (22)
| Country | Link |
|---|---|
| US (1) | US20150284472A1 (enExample) |
| EP (1) | EP2914281A1 (enExample) |
| JP (1) | JP2016503405A (enExample) |
| KR (1) | KR20150079751A (enExample) |
| CN (1) | CN104902923A (enExample) |
| AR (1) | AR093355A1 (enExample) |
| AU (1) | AU2013337354A1 (enExample) |
| BR (1) | BR112015009746A2 (enExample) |
| CA (1) | CA2889990A1 (enExample) |
| CL (1) | CL2015001157A1 (enExample) |
| CR (1) | CR20150216A (enExample) |
| EA (1) | EA201590880A1 (enExample) |
| HK (1) | HK1214521A1 (enExample) |
| IL (1) | IL238416A0 (enExample) |
| MA (1) | MA38144A1 (enExample) |
| MX (1) | MX2015005722A (enExample) |
| PH (1) | PH12015500879A1 (enExample) |
| SG (1) | SG11201502989XA (enExample) |
| TN (1) | TN2015000171A1 (enExample) |
| TW (1) | TW201427695A (enExample) |
| WO (1) | WO2014071282A1 (enExample) |
| ZA (1) | ZA201502618B (enExample) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK3578195T5 (da) | 2008-06-26 | 2024-06-10 | Zevra Denmark As | Anvendelse af Hsp70 som regulator af enzymatisk aktivitet |
| WO2012072082A1 (en) | 2010-11-30 | 2012-06-07 | Orphazyme Aps | Methods for increasing intracellular activity of hsp70 |
| HUE054957T2 (hu) | 2014-09-15 | 2021-10-28 | Orphazyme As | Arimoklomol készítése |
| CA2985235A1 (en) * | 2015-05-07 | 2016-11-10 | Shire Human Genetic Therapies, Inc. | Glucocerebrosidase gene therapy for parkinson's disease |
| WO2017015491A1 (en) * | 2015-07-21 | 2017-01-26 | Thomas Jefferson University | Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways |
| WO2017127679A1 (en) * | 2016-01-21 | 2017-07-27 | Protein Dynamic Solutions Llc | Method and system for spectral data analysis |
| WO2017178029A1 (en) | 2016-04-13 | 2017-10-19 | Orphazyme Aps | Heat shock proteins and cholesterol homeostasis |
| CA3287846A1 (en) * | 2016-04-29 | 2025-11-29 | Zevra Denmark A/S | Arimoclomol for treating glucocerebrosidase associated disorders |
| EP3264092A1 (en) * | 2016-07-01 | 2018-01-03 | Centogene AG | Use of lyso-gb1 as druggable target |
| KR102523237B1 (ko) * | 2016-08-03 | 2023-04-18 | 유니버시티 오브 사우스 플로리다 | 신경 장애의 치료를 위한 리일린 조성물 |
| WO2019070891A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISORDERS |
| KR102700963B1 (ko) | 2017-10-03 | 2024-09-02 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| IL278868B2 (en) * | 2017-10-03 | 2025-06-01 | Prevail Therapeutics Inc | Gene therapies for lysosomal disorders |
| EP3701030A4 (en) * | 2017-10-23 | 2022-04-20 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR NEURODEGENERATIVE DISEASES |
| AR113797A1 (es) * | 2017-10-26 | 2020-06-10 | Shire Human Genetic Therapies | Formulaciones que comprenden glucocerebrosidasa e isofagomina |
| EP3765855A1 (en) * | 2018-03-14 | 2021-01-20 | Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd | Detection of phospho-serine 129 alpha-synuclein in blood cells as a biomarker for synucleinopathies |
| US11999974B2 (en) | 2019-04-10 | 2024-06-04 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| MX2021012184A (es) * | 2019-04-10 | 2022-01-24 | Prevail Therapeutics Inc | Terapias genicas para trastornos lisosomales. |
| EP3986412A4 (en) | 2019-06-21 | 2023-11-15 | The Broad Institute, Inc. | Agents for reversing toxic proteinopathies |
| CA3181024A1 (en) | 2020-08-06 | 2022-02-10 | Fundacion Para La Investigacion Medica Aplicada | Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy |
| CN116420078A (zh) * | 2020-10-14 | 2023-07-11 | 戴纳立制药公司 | 用于治疗和监测额颞痴呆的方法 |
| KR20230128462A (ko) | 2020-11-19 | 2023-09-05 | 제브라 덴마크 에이/에스 | 아리모클로몰 시트레이트 및 이의 중간체를 제조하기 위한 공정 |
| CN112569354B (zh) * | 2020-12-29 | 2022-06-10 | 四川大学华西医院 | tau蛋白及其基因作为药物靶点在制备治疗糖尿病药物中的应用 |
| JP2024546925A (ja) * | 2021-12-17 | 2024-12-26 | エフ. ホフマン-ラ ロシュ アーゲー | グルコセレブロシダーゼ発現を増加させることができるオリゴヌクレオチド |
| EP4448763A1 (en) * | 2021-12-17 | 2024-10-23 | F. Hoffmann-La Roche AG | Oligonucleotide gba agonists |
Family Cites Families (33)
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| US4816567A (en) | 1983-04-08 | 1989-03-28 | Genentech, Inc. | Recombinant immunoglobin preparations |
| US4683195A (en) | 1986-01-30 | 1987-07-28 | Cetus Corporation | Process for amplifying, detecting, and/or-cloning nucleic acid sequences |
| US4676980A (en) | 1985-09-23 | 1987-06-30 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Target specific cross-linked heteroantibodies |
| US5225539A (en) | 1986-03-27 | 1993-07-06 | Medical Research Council | Recombinant altered antibodies and methods of making altered antibodies |
| US5892019A (en) | 1987-07-15 | 1999-04-06 | The United States Of America, As Represented By The Department Of Health And Human Services | Production of a single-gene-encoded immunoglobulin |
| GB8823869D0 (en) | 1988-10-12 | 1988-11-16 | Medical Res Council | Production of antibodies |
| US6172197B1 (en) | 1991-07-10 | 2001-01-09 | Medical Research Council | Methods for producing members of specific binding pairs |
| GB9015198D0 (en) | 1990-07-10 | 1990-08-29 | Brien Caroline J O | Binding substance |
| ES2108048T3 (es) | 1990-08-29 | 1997-12-16 | Genpharm Int | Produccion y utilizacion de animales inferiores transgenicos capaces de producir anticuerpos heterologos. |
| US5633425A (en) | 1990-08-29 | 1997-05-27 | Genpharm International, Inc. | Transgenic non-human animals capable of producing heterologous antibodies |
| US5661016A (en) | 1990-08-29 | 1997-08-26 | Genpharm International Inc. | Transgenic non-human animals capable of producing heterologous antibodies of various isotypes |
| US5625126A (en) | 1990-08-29 | 1997-04-29 | Genpharm International, Inc. | Transgenic non-human animals for producing heterologous antibodies |
| US5545806A (en) | 1990-08-29 | 1996-08-13 | Genpharm International, Inc. | Ransgenic non-human animals for producing heterologous antibodies |
| DE69129154T2 (de) | 1990-12-03 | 1998-08-20 | Genentech, Inc., South San Francisco, Calif. | Verfahren zur anreicherung von proteinvarianten mit geänderten bindungseigenschaften |
| ATE463573T1 (de) | 1991-12-02 | 2010-04-15 | Medimmune Ltd | Herstellung von autoantikörpern auf phagenoberflächen ausgehend von antikörpersegmentbibliotheken |
| ES2249761T3 (es) | 1993-06-24 | 2006-04-01 | Advec Inc. | Vectores de adenovirus para terapia genica. |
| SK283703B6 (sk) | 1993-10-25 | 2003-12-02 | Canji, Inc. | Rekombinantný adenovírusový vektor a jeho použitie |
| US7252989B1 (en) | 1994-04-04 | 2007-08-07 | Board Of Regents, The University Of Texas System | Adenovirus supervector system |
| US6828422B1 (en) | 1995-08-18 | 2004-12-07 | Morphosys Ag | Protein/(poly)peptide libraries |
| WO1997008320A1 (en) | 1995-08-18 | 1997-03-06 | Morphosys Gesellschaft Für Proteinoptimierung Mbh | Protein/(poly)peptide libraries |
| US6924123B2 (en) | 1996-10-29 | 2005-08-02 | Oxford Biomedica (Uk) Limited | Lentiviral LTR-deleted vector |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| WO2001002588A2 (en) | 1999-07-02 | 2001-01-11 | Morphosys Ag | Generation of specific binding partners binding to (poly)peptides encoded by genomic dna fragments or ests |
| ATE403715T1 (de) | 1999-08-09 | 2008-08-15 | Targeted Genetics Corp | Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| NZ522840A (en) | 2000-06-01 | 2004-12-24 | Univ North Carolina | A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together |
| WO2001097829A2 (en) | 2000-06-19 | 2001-12-27 | Genzyme Corporation | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| US7446098B2 (en) * | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
| US7765583B2 (en) | 2005-02-28 | 2010-07-27 | France Telecom | System and method for managing virtual user domains |
| JP5303458B2 (ja) * | 2006-06-23 | 2013-10-02 | アミークス セラピューティックス インコーポレイテッド | β−グルコセレブロシダーゼの活性増強による神経学的疾患の治療方法 |
| ES2559859T3 (es) * | 2007-05-16 | 2016-02-16 | The Brigham And Women's Hospital, Inc. | Tratamiento de sinucleinopatías |
| CN103764166B (zh) | 2011-06-22 | 2017-10-24 | 通用医疗公司 | 蛋白质病的治疗 |
| WO2013148333A1 (en) | 2012-03-28 | 2013-10-03 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Salicylic acid derivatives useful as glucocerebrosidase activators |
-
2013
- 2013-11-04 EA EA201590880A patent/EA201590880A1/ru unknown
- 2013-11-04 WO PCT/US2013/068242 patent/WO2014071282A1/en not_active Ceased
- 2013-11-04 US US14/440,818 patent/US20150284472A1/en not_active Abandoned
- 2013-11-04 AU AU2013337354A patent/AU2013337354A1/en not_active Abandoned
- 2013-11-04 SG SG11201502989XA patent/SG11201502989XA/en unknown
- 2013-11-04 CN CN201380069582.2A patent/CN104902923A/zh active Pending
- 2013-11-04 MX MX2015005722A patent/MX2015005722A/es unknown
- 2013-11-04 CA CA2889990A patent/CA2889990A1/en not_active Abandoned
- 2013-11-04 HK HK16102536.9A patent/HK1214521A1/zh unknown
- 2013-11-04 MA MA38144A patent/MA38144A1/fr unknown
- 2013-11-04 EP EP13789690.8A patent/EP2914281A1/en not_active Withdrawn
- 2013-11-04 BR BR112015009746A patent/BR112015009746A2/pt not_active IP Right Cessation
- 2013-11-04 JP JP2015540845A patent/JP2016503405A/ja not_active Abandoned
- 2013-11-04 TW TW102139891A patent/TW201427695A/zh unknown
- 2013-11-04 KR KR1020157013541A patent/KR20150079751A/ko not_active Withdrawn
- 2013-11-05 AR ARP130104033A patent/AR093355A1/es unknown
-
2015
- 2015-04-17 ZA ZA2015/02618A patent/ZA201502618B/en unknown
- 2015-04-20 PH PH12015500879A patent/PH12015500879A1/en unknown
- 2015-04-21 IL IL238416A patent/IL238416A0/en unknown
- 2015-04-28 CR CR20150216A patent/CR20150216A/es unknown
- 2015-04-30 CL CL2015001157A patent/CL2015001157A1/es unknown
- 2015-05-04 TN TNP2015000171A patent/TN2015000171A1/fr unknown
Also Published As
| Publication number | Publication date |
|---|---|
| WO2014071282A1 (en) | 2014-05-08 |
| SG11201502989XA (en) | 2015-05-28 |
| IL238416A0 (en) | 2015-06-30 |
| PH12015500879A1 (en) | 2015-06-29 |
| KR20150079751A (ko) | 2015-07-08 |
| CA2889990A1 (en) | 2014-05-08 |
| JP2016503405A (ja) | 2016-02-04 |
| AR093355A1 (es) | 2015-06-03 |
| TW201427695A (zh) | 2014-07-16 |
| HK1214521A1 (zh) | 2016-07-29 |
| ZA201502618B (en) | 2016-01-27 |
| EA201590880A1 (ru) | 2015-09-30 |
| CN104902923A (zh) | 2015-09-09 |
| CR20150216A (es) | 2015-05-29 |
| CL2015001157A1 (es) | 2015-10-16 |
| AU2013337354A1 (en) | 2015-05-21 |
| TN2015000171A1 (fr) | 2016-10-03 |
| EP2914281A1 (en) | 2015-09-09 |
| MA38144A1 (fr) | 2018-08-31 |
| US20150284472A1 (en) | 2015-10-08 |
| MX2015005722A (es) | 2016-01-12 |
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