WO1996009074B1 - Utilisation d'un virus a adn non mammalien en vue de l'expression d'un gene exogene dans une cellule mammalienne - Google Patents
Utilisation d'un virus a adn non mammalien en vue de l'expression d'un gene exogene dans une cellule mammalienneInfo
- Publication number
- WO1996009074B1 WO1996009074B1 PCT/US1995/011456 US9511456W WO9609074B1 WO 1996009074 B1 WO1996009074 B1 WO 1996009074B1 US 9511456 W US9511456 W US 9511456W WO 9609074 B1 WO9609074 B1 WO 9609074B1
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- promoters
- genome
- promoter
- gene
- nucleic acid
- Prior art date
Links
- 108090000623 proteins and genes Proteins 0.000 title claims abstract 44
- 241000700605 Viruses Species 0.000 title claims abstract 23
- 210000004962 mammalian cell Anatomy 0.000 title abstract 3
- 210000004027 cell Anatomy 0.000 claims abstract 34
- 238000000034 method Methods 0.000 claims abstract 13
- 241000701447 unidentified baculovirus Species 0.000 claims abstract 10
- 241000124008 Mammalia Species 0.000 claims abstract 4
- 230000007812 deficiency Effects 0.000 claims abstract 2
- 108020004707 nucleic acids Proteins 0.000 claims 30
- 102000039446 nucleic acids Human genes 0.000 claims 30
- 150000007523 nucleic acids Chemical class 0.000 claims 30
- 108020004414 DNA Proteins 0.000 claims 8
- 241000238631 Hexapoda Species 0.000 claims 7
- 108090000364 Ligases Proteins 0.000 claims 6
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- NOESYZHRGYRDHS-UHFFFAOYSA-N insulin Chemical compound N1C(=O)C(NC(=O)C(CCC(N)=O)NC(=O)C(CCC(O)=O)NC(=O)C(C(C)C)NC(=O)C(NC(=O)CN)C(C)CC)CSSCC(C(NC(CO)C(=O)NC(CC(C)C)C(=O)NC(CC=2C=CC(O)=CC=2)C(=O)NC(CCC(N)=O)C(=O)NC(CC(C)C)C(=O)NC(CCC(O)=O)C(=O)NC(CC(N)=O)C(=O)NC(CC=2C=CC(O)=CC=2)C(=O)NC(CSSCC(NC(=O)C(C(C)C)NC(=O)C(CC(C)C)NC(=O)C(CC=2C=CC(O)=CC=2)NC(=O)C(CC(C)C)NC(=O)C(C)NC(=O)C(CCC(O)=O)NC(=O)C(C(C)C)NC(=O)C(CC(C)C)NC(=O)C(CC=2NC=NC=2)NC(=O)C(CO)NC(=O)CNC2=O)C(=O)NCC(=O)NC(CCC(O)=O)C(=O)NC(CCCNC(N)=N)C(=O)NCC(=O)NC(CC=3C=CC=CC=3)C(=O)NC(CC=3C=CC=CC=3)C(=O)NC(CC=3C=CC(O)=CC=3)C(=O)NC(C(C)O)C(=O)N3C(CCC3)C(=O)NC(CCCCN)C(=O)NC(C)C(O)=O)C(=O)NC(CC(N)=O)C(O)=O)=O)NC(=O)C(C(C)CC)NC(=O)C(CO)NC(=O)C(C(C)O)NC(=O)C1CSSCC2NC(=O)C(CC(C)C)NC(=O)C(NC(=O)C(CCC(N)=O)NC(=O)C(CC(N)=O)NC(=O)C(NC(=O)C(N)CC=1C=CC=CC=1)C(C)C)CC1=CN=CN1 NOESYZHRGYRDHS-UHFFFAOYSA-N 0.000 claims 6
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- 102000014750 Phosphorylase Kinase Human genes 0.000 claims 3
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- 102100034391 Porphobilinogen deaminase Human genes 0.000 claims 3
- 108010053763 Pyruvate Carboxylase Proteins 0.000 claims 3
- 102100039895 Pyruvate carboxylase, mitochondrial Human genes 0.000 claims 3
- 102000006601 Thymidine Kinase Human genes 0.000 claims 3
- 208000018839 Wilson disease Diseases 0.000 claims 3
- 102000006995 beta-Glucosidase Human genes 0.000 claims 3
- 108010047754 beta-Glucosidase Proteins 0.000 claims 3
- 125000003917 carbamoyl group Chemical group [H]N([H])C(*)=O 0.000 claims 3
- ILRYLPWNYFXEMH-UHFFFAOYSA-N cystathionine Chemical compound OC(=O)C(N)CCSCC(N)C(O)=O ILRYLPWNYFXEMH-UHFFFAOYSA-N 0.000 claims 3
- 229960004222 factor ix Drugs 0.000 claims 3
- 108010022687 fumarylacetoacetase Proteins 0.000 claims 3
- 230000002440 hepatic effect Effects 0.000 claims 3
- 229940125396 insulin Drugs 0.000 claims 3
- 210000004185 liver Anatomy 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 241000701161 unidentified adenovirus Species 0.000 claims 3
- 241001430294 unidentified retrovirus Species 0.000 claims 3
- 201000009030 Carcinoma Diseases 0.000 claims 2
- 241000701022 Cytomegalovirus Species 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 108010053187 Diphtheria Toxin Proteins 0.000 claims 2
- 108010054218 Factor VIII Proteins 0.000 claims 2
- 102000001690 Factor VIII Human genes 0.000 claims 2
- 102000003638 Glucose-6-Phosphatase Human genes 0.000 claims 2
- 108010086800 Glucose-6-Phosphatase Proteins 0.000 claims 2
- 108010015451 Glutaryl-CoA Dehydrogenase Proteins 0.000 claims 2
- 102100028603 Glutaryl-CoA dehydrogenase, mitochondrial Human genes 0.000 claims 2
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 claims 2
- 102000004327 Glycine dehydrogenase (decarboxylating) Human genes 0.000 claims 2
- 108090000826 Glycine dehydrogenase (decarboxylating) Proteins 0.000 claims 2
- 102000014150 Interferons Human genes 0.000 claims 2
- 108010050904 Interferons Proteins 0.000 claims 2
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 claims 2
- 108010085747 Methylmalonyl-CoA Decarboxylase Proteins 0.000 claims 2
- 206010028980 Neoplasm Diseases 0.000 claims 2
- 108010035004 Prephenate Dehydrogenase Proteins 0.000 claims 2
- 241000700584 Simplexvirus Species 0.000 claims 2
- 108091023040 Transcription factor Proteins 0.000 claims 2
- 102000040945 Transcription factor Human genes 0.000 claims 2
- 108060008682 Tumor Necrosis Factor Proteins 0.000 claims 2
- 108010026331 alpha-Fetoproteins Proteins 0.000 claims 2
- 102000013529 alpha-Fetoproteins Human genes 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- 229940104302 cytosine Drugs 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 229960000301 factor viii Drugs 0.000 claims 2
- 229940012952 fibrinogen Drugs 0.000 claims 2
- 239000003102 growth factor Substances 0.000 claims 2
- 230000001939 inductive effect Effects 0.000 claims 2
- 229940047124 interferons Drugs 0.000 claims 2
- 150000004715 keto acids Chemical class 0.000 claims 2
- 230000006269 (delayed) early viral mRNA transcription Effects 0.000 claims 1
- 108020004774 Alkaline Phosphatase Proteins 0.000 claims 1
- 102100022524 Alpha-1-antichymotrypsin Human genes 0.000 claims 1
- 102100033312 Alpha-2-macroglobulin Human genes 0.000 claims 1
- 108020005544 Antisense RNA Proteins 0.000 claims 1
- 241001203868 Autographa californica Species 0.000 claims 1
- 108090000994 Catalytic RNA Proteins 0.000 claims 1
- 108010075016 Ceruloplasmin Proteins 0.000 claims 1
- 102100023321 Ceruloplasmin Human genes 0.000 claims 1
- 108010035563 Chloramphenicol O-acetyltransferase Proteins 0.000 claims 1
- 102000000989 Complement System Proteins Human genes 0.000 claims 1
- 108010069112 Complement System Proteins Proteins 0.000 claims 1
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- 239000004471 Glycine Substances 0.000 claims 1
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- 102000012404 Orosomucoid Human genes 0.000 claims 1
- 102100038124 Plasminogen Human genes 0.000 claims 1
- 108010051456 Plasminogen Proteins 0.000 claims 1
- 108010071690 Prealbumin Proteins 0.000 claims 1
- 108010015078 Pregnancy-Associated alpha 2-Macroglobulins Proteins 0.000 claims 1
- LCTONWCANYUPML-UHFFFAOYSA-M Pyruvate Chemical compound CC(=O)C([O-])=O LCTONWCANYUPML-UHFFFAOYSA-M 0.000 claims 1
- 102000013009 Pyruvate Kinase Human genes 0.000 claims 1
- 108020005115 Pyruvate Kinase Proteins 0.000 claims 1
- 102000004338 Transferrin Human genes 0.000 claims 1
- 108090000901 Transferrin Proteins 0.000 claims 1
- 102000009190 Transthyretin Human genes 0.000 claims 1
- 239000012190 activator Substances 0.000 claims 1
- 108010091628 alpha 1-Antichymotrypsin Proteins 0.000 claims 1
- 239000012830 cancer therapeutic Substances 0.000 claims 1
- 239000003184 complementary RNA Substances 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- -1 factor vni Proteins 0.000 claims 1
- 208000005252 hepatitis A Diseases 0.000 claims 1
- 208000002672 hepatitis B Diseases 0.000 claims 1
- 206010073071 hepatocellular carcinoma Diseases 0.000 claims 1
- 231100000844 hepatocellular carcinoma Toxicity 0.000 claims 1
- 210000003494 hepatocyte Anatomy 0.000 claims 1
- 210000005260 human cell Anatomy 0.000 claims 1
- 210000003125 jurkat cell Anatomy 0.000 claims 1
- 210000003292 kidney cell Anatomy 0.000 claims 1
- 101150066555 lacZ gene Proteins 0.000 claims 1
- 210000005265 lung cell Anatomy 0.000 claims 1
- 210000003098 myoblast Anatomy 0.000 claims 1
- 102000042567 non-coding RNA Human genes 0.000 claims 1
- LCCNCVORNKJIRZ-UHFFFAOYSA-N parathion Chemical compound CCOP(=S)(OCC)OC1=CC=C([N+]([O-])=O)C=C1 LCCNCVORNKJIRZ-UHFFFAOYSA-N 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- QAQREVBBADEHPA-IEXPHMLFSA-N propionyl-CoA Chemical compound O[C@@H]1[C@H](OP(O)(O)=O)[C@@H](COP(O)(=O)OP(O)(=O)OCC(C)(C)[C@@H](O)C(=O)NCCC(=O)NCCSC(=O)CC)O[C@H]1N1C2=NC=NC(N)=C2N=C1 QAQREVBBADEHPA-IEXPHMLFSA-N 0.000 claims 1
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- 230000001225 therapeutic effect Effects 0.000 claims 1
- 239000012581 transferrin Substances 0.000 claims 1
- 102000003390 tumor necrosis factor Human genes 0.000 claims 1
- 241000701366 unidentified nuclear polyhedrosis viruses Species 0.000 claims 1
Abstract
L'invention a pour objet un procédé d'expression d'un gène exogène dans une cellule mammalienne. Le procédé consiste à infecter la cellule à l'aide d'un virus non mammalien (par exemple un baculovirus) dont le génome est porteur d'un gène exogène, et à cultiver la cellule dans des conditions permettant l'expression du gène. Les gènes exogènes sont introduits dans les cellules mammaliennes à l'aide d'un vecteur de transfert tel que celui représenté dans la figure. L'invention concerne également un procédé permettant de traiter un trouble lié à une carence génique chez un mammifère. On introduit dans une cellule une quantité thérapeutique efficace d'un virus dont le génome est porteur d'un gène exogène, et on cultive ladite cellule dans des conditions permettant l'expression du gène exogène chez un mammifère.
Priority Applications (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP95934407A EP0785803A4 (fr) | 1994-09-23 | 1995-09-08 | Utilisation d'un virus a adn non mammalien en vue de l'expression d'un gene exogene dans une cellule mammalienne |
| AU36750/95A AU702830B2 (en) | 1994-09-23 | 1995-09-08 | Use of a non-mammalian dna virus to express an exogenous gene in a mammalian cell |
| JP8510940A JPH10506530A (ja) | 1994-09-23 | 1995-09-08 | 哺乳類細胞において外来遺伝子を発現する非哺乳類ｄｎａウイルスの使用 |
| MXPA/A/1997/002132A MXPA97002132A (en) | 1994-09-23 | 1997-03-20 | Use of a non-myamiferous dna virus to express an exogenous gene in a mamif cell |
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US08/311,157 | 1994-09-23 | ||
| US08/311,157 US5871986A (en) | 1994-09-23 | 1994-09-23 | Use of a baculovirus to express and exogenous gene in a mammalian cell |
| US08/486,341 US5731182A (en) | 1994-09-23 | 1995-06-07 | Non-mammalian DNA virus to express an exogenous gene in a mammalian cell |
| US08/486,341 | 1995-06-07 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| WO1996009074A1 WO1996009074A1 (fr) | 1996-03-28 |
| WO1996009074B1 true WO1996009074B1 (fr) | 1996-05-09 |
Family
ID=26977767
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PCT/US1995/011456 WO1996009074A1 (fr) | 1994-09-23 | 1995-09-08 | Utilisation d'un virus a adn non mammalien en vue de l'expression d'un gene exogene dans une cellule mammalienne |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US6238914B1 (fr) |
| EP (1) | EP0785803A4 (fr) |
| JP (1) | JPH10506530A (fr) |
| CN (1) | CN1172435A (fr) |
| AU (1) | AU702830B2 (fr) |
| CA (1) | CA2200835A1 (fr) |
| IL (1) | IL115267A0 (fr) |
| OA (1) | OA10408A (fr) |
| TW (1) | TW464500B (fr) |
| WO (1) | WO1996009074A1 (fr) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5670347A (en) | 1994-05-11 | 1997-09-23 | Amba Biosciences Llc | Peptide-mediated gene transfer |
| US5871986A (en) | 1994-09-23 | 1999-02-16 | The General Hospital Corporation | Use of a baculovirus to express and exogenous gene in a mammalian cell |
| US6183993B1 (en) | 1996-09-11 | 2001-02-06 | The General Hospital Corporation | Complement-resistant non-mammalian DNA viruses and uses thereof |
| BR9712978A (pt) * | 1996-09-11 | 2000-04-18 | Gen Hospital Corp | Vìrus de dna de não-mamìferos possuindo uma proteìna de revestimento alterada |
| WO1998012311A2 (fr) * | 1996-09-11 | 1998-03-26 | The General Hospital Corporation | Utilisation d'un virus a adn non mammalien pour l'expression d'un gene exogene dans une cellule mammalienne |
| FR2756297B1 (fr) | 1996-11-22 | 1999-01-08 | Centre Nat Rech Scient | Procede de production de virus recombinants |
| IT1291135B1 (it) * | 1997-04-08 | 1998-12-29 | Angeletti P Ist Richerche Bio | Vettori ricombinanti utilizzabili in terapia genica |
| FR2761994B1 (fr) | 1997-04-11 | 1999-06-18 | Centre Nat Rech Scient | Preparation de recepteurs membranaires a partir de baculovirus extracellulaires |
| GB9708698D0 (en) * | 1997-04-29 | 1997-06-18 | Imperial College | Materials for therapy |
| US6106825A (en) * | 1997-05-07 | 2000-08-22 | University Of Florida | Entomopoxvirus-vertebrate gene delivery vector and method |
| DE19735593C2 (de) * | 1997-08-15 | 1999-08-26 | Hepavec Ag Fuer Gentherapie | Hüllprotein-modifizierter Baculovirus-Vektor für die Gentherapie |
| US6346415B1 (en) * | 1997-10-21 | 2002-02-12 | Targeted Genetics Corporation | Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors |
| US6933145B1 (en) | 1998-05-29 | 2005-08-23 | University Of Florida Research Foundation, Inc. | Materials and methods for delivery and expression of heterologous DNA in vertebrate cells |
| GB0119852D0 (en) * | 2001-08-15 | 2001-10-10 | Univ York | Baculovirus |
| WO2003020322A1 (fr) * | 2001-09-04 | 2003-03-13 | DeveloGen Aktiengesellschaft für entwicklungsbiologische Forschung | Vecteur d'induction d'une reponse immune |
| TWI288779B (en) * | 2002-03-28 | 2007-10-21 | Blasticon Biotech Forschung | Dedifferentiated, programmable stem cells of monocytic origin, and their production and use |
| JP2003286199A (ja) * | 2002-03-29 | 2003-10-07 | Japan Science & Technology Corp | タンパク質中空ナノ粒子を用いる肝臓疾患治療用薬剤 |
| JP2003286198A (ja) * | 2002-03-29 | 2003-10-07 | Japan Science & Technology Corp | 増殖因子等を提示するタンパク質中空ナノ粒子を用いる治療薬剤 |
| CA2500661A1 (fr) * | 2002-10-01 | 2004-04-15 | Chiron Corporation | Compositions anticancereuses et contre les maladies infectieuses, et procedes d'utilisation correspondants |
| GB0425739D0 (en) * | 2004-11-23 | 2004-12-22 | Procure Therapeutics Ltd | Humanised baculovirus 2 |
| US20090055941A1 (en) * | 2005-03-22 | 2009-02-26 | Agency For Science, Technology And Research | Novel Neural Cell Specific Promoter And Baculovirus And Method For Gene Delivery |
| US20080044855A1 (en) * | 2006-03-22 | 2008-02-21 | Yan Xu | System for regulated and enhanced baculovirus mediated transient transgene expression in mammalian cells |
| FR2966841A1 (fr) * | 2010-11-02 | 2012-05-04 | Inst Nat De Rech Agronomique Inra | Vecteurs derives de densovirus pour le transfert de gene chez l'insecte |
| CN102199629A (zh) * | 2011-03-29 | 2011-09-28 | 中国科学院武汉病毒研究所 | 用于提高杆状病毒对哺乳动物细胞转导效率的方法 |
| US9616114B1 (en) | 2014-09-18 | 2017-04-11 | David Gordon Bermudes | Modified bacteria having improved pharmacokinetics and tumor colonization enhancing antitumor activity |
| US10676723B2 (en) | 2015-05-11 | 2020-06-09 | David Gordon Bermudes | Chimeric protein toxins for expression by therapeutic bacteria |
| US11180535B1 (en) | 2016-12-07 | 2021-11-23 | David Gordon Bermudes | Saccharide binding, tumor penetration, and cytotoxic antitumor chimeric peptides from therapeutic bacteria |
| US11129906B1 (en) | 2016-12-07 | 2021-09-28 | David Gordon Bermudes | Chimeric protein toxins for expression by therapeutic bacteria |
| US10842853B2 (en) | 2017-05-22 | 2020-11-24 | Baxalta Incorporated | Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia B |
| US10995345B2 (en) | 2017-09-12 | 2021-05-04 | Biocapital Holdings, Llc | Biological devices for producing oxidized zinc and applications thereof |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| PE20212076A1 (es) | 2018-02-01 | 2021-10-26 | Homology Medicines Inc | Composiciones de virus adeno-asociado para restaurar la funcion del gen pah y metodos de uso de las mismas |
| US11306329B2 (en) | 2018-02-19 | 2022-04-19 | City Of Hope | Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof |
| US11298414B2 (en) | 2018-08-10 | 2022-04-12 | Nantbio, Inc. | Cellular adjuvants for viral infection |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4745051A (en) * | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| US4879236A (en) * | 1984-05-16 | 1989-11-07 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| FR2579224B1 (fr) * | 1985-03-25 | 1987-05-22 | Genetica | Procede de preparation microbiologique de la serum-albumine humaine |
| US5004687A (en) * | 1985-05-21 | 1991-04-02 | University Of Georgia Research Foundation, Inc. | Insect virus vector with broadened host range |
| US5106741A (en) * | 1985-12-20 | 1992-04-21 | The Upjohn Company | Tissue plasminogen activator (TPA) analogs |
| US5476781A (en) * | 1991-02-19 | 1995-12-19 | University Of Florida Research Foundation, Inc. | Entomopoxvirus spheroidin gene sequences |
| DE4407859C1 (de) * | 1994-03-04 | 1995-03-02 | Max Planck Gesellschaft | Vektor für die leberspezifische Gentherapie |
| US5656465A (en) * | 1994-05-04 | 1997-08-12 | Therion Biologics Corporation | Methods of in vivo gene delivery |
| US5871986A (en) * | 1994-09-23 | 1999-02-16 | The General Hospital Corporation | Use of a baculovirus to express and exogenous gene in a mammalian cell |
-
1995
- 1995-09-08 EP EP95934407A patent/EP0785803A4/fr not_active Withdrawn
- 1995-09-08 WO PCT/US1995/011456 patent/WO1996009074A1/fr not_active Application Discontinuation
- 1995-09-08 CN CN95196379A patent/CN1172435A/zh active Pending
- 1995-09-08 JP JP8510940A patent/JPH10506530A/ja active Pending
- 1995-09-08 CA CA002200835A patent/CA2200835A1/fr not_active Abandoned
- 1995-09-08 AU AU36750/95A patent/AU702830B2/en not_active Ceased
- 1995-09-12 IL IL11526795A patent/IL115267A0/xx unknown
- 1995-09-23 TW TW084109934A patent/TW464500B/zh not_active IP Right Cessation
-
1996
- 1996-11-19 US US08/752,030 patent/US6238914B1/en not_active Expired - Lifetime
-
1997
- 1997-03-21 OA OA60981A patent/OA10408A/en unknown
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