US20100190689A1 - Compositions and methods related to protein displacement therapy for myotonic distrophy - Google Patents

Compositions and methods related to protein displacement therapy for myotonic distrophy Download PDF

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Publication number
US20100190689A1
US20100190689A1 US12/442,354 US44235407A US2010190689A1 US 20100190689 A1 US20100190689 A1 US 20100190689A1 US 44235407 A US44235407 A US 44235407A US 2010190689 A1 US2010190689 A1 US 2010190689A1
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Prior art keywords
protein
agent
mbnl1
labeled
morpholino
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Abandoned
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US12/442,354
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English (en)
Inventor
Charles A. Thornton
Thurman Wheeler
Krzysztof Sobezak
Robert Osborne
Jill Miller
Maurice Swanson
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University of Florida Research Foundation Inc
University of Rochester
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University of Florida Research Foundation Inc
University of Rochester
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Priority to US12/442,354 priority Critical patent/US20100190689A1/en
Assigned to UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC. reassignment UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC. ASSIGNMENT OF ASSIGNORS INTEREST (SEE DOCUMENT FOR DETAILS). Assignors: SWANSON, MAURICE SCOTT
Publication of US20100190689A1 publication Critical patent/US20100190689A1/en
Assigned to NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT reassignment NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT CONFIRMATORY LICENSE (SEE DOCUMENT FOR DETAILS). Assignors: UNIVERSITY OF ROCHESTER
Assigned to UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC. reassignment UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC. ASSIGNMENT OF ASSIGNORS INTEREST (SEE DOCUMENT FOR DETAILS). Assignors: SWANSON, MAURICE SCOTT
Assigned to UNIVERSITY OF ROCHESTER reassignment UNIVERSITY OF ROCHESTER ASSIGNMENT OF ASSIGNORS INTEREST (SEE DOCUMENT FOR DETAILS). Assignors: WHEELER, THURMAN, THORNTON, CHARLES A., MILLER, JILL
Assigned to NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT reassignment NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT CONFIRMATORY LICENSE (SEE DOCUMENT FOR DETAILS). Assignors: UNIVERSITY OF ROCHESTER
Abandoned legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • 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
    • A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
    • A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
    • A61K31/7036—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin having at least one amino group directly attached to the carbocyclic ring, e.g. streptomycin, gentamycin, amikacin, validamycin, fortimicins
    • 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
    • A61K47/54—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 an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6872—Intracellular protein regulatory factors and their receptors, e.g. including ion channels
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6887—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids from muscle, cartilage or connective tissue
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2500/00—Screening for compounds of potential therapeutic value
    • G01N2500/02—Screening involving studying the effect of compounds C on the interaction between interacting molecules A and B (e.g. A = enzyme and B = substrate for A, or A = receptor and B = ligand for the receptor)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders
    • G01N2800/2878—Muscular dystrophy
US12/442,354 2006-09-21 2007-09-21 Compositions and methods related to protein displacement therapy for myotonic distrophy Abandoned US20100190689A1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
US12/442,354 US20100190689A1 (en) 2006-09-21 2007-09-21 Compositions and methods related to protein displacement therapy for myotonic distrophy

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US82639606P 2006-09-21 2006-09-21
PCT/US2007/020503 WO2008036406A2 (fr) 2006-09-21 2007-09-21 Compositions et procédés se rapportant à une thérapie par glissement de protéines pour la distrophie myotonique
US12/442,354 US20100190689A1 (en) 2006-09-21 2007-09-21 Compositions and methods related to protein displacement therapy for myotonic distrophy

Related Parent Applications (1)

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PCT/US2007/020503 A-371-Of-International WO2008036406A2 (fr) 2006-09-21 2007-09-21 Compositions et procédés se rapportant à une thérapie par glissement de protéines pour la distrophie myotonique

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US14/297,137 Active US9371527B2 (en) 2006-09-21 2014-06-05 Compositions and methods related to protein displacement therapy for myotonic distrophy

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US (2) US20100190689A1 (fr)
EP (3) EP2560001B1 (fr)
JP (3) JP5926475B2 (fr)
AU (1) AU2007297535C1 (fr)
CA (2) CA2981308C (fr)
WO (1) WO2008036406A2 (fr)

Cited By (13)

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US20090082547A1 (en) * 2003-04-29 2009-03-26 Iversen Patrick L Compositions for enhancing transport of molecules into cells
US20140134737A1 (en) * 2011-06-30 2014-05-15 Governing Council Of The University Of Toronto Alternative Splicing Modulators and Splice Variants and Their Use in the Control and Detection of Pluripotency and Differentiation
US8741863B2 (en) 2007-06-29 2014-06-03 Sarepta Therapeutics, Inc. Compound and method for treating myotonic dystrophy
US8835402B2 (en) 2009-06-26 2014-09-16 Sarepta Therapeutics, Inc. Compound and method for treating myotonic dystrophy
WO2014171698A1 (fr) * 2013-04-18 2014-10-23 사회복지법인 삼성생명공익재단 Méthode de diagnostic de la dystrophie myotonique de type 1
US8877725B2 (en) 2004-05-24 2014-11-04 Sarepta Therapeutics, Inc. Peptide conjugated, inosine-substituted antisense oligomer compound and method
US9075055B2 (en) 2013-03-15 2015-07-07 Hycor Biomedical, Inc. Device and associated methods for performing luminescence and fluorescence measurements of a sample
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
US9499583B2 (en) 2005-07-13 2016-11-22 Sarepta Therapeutics, Inc. Antibacterial antisense oligonucleotide and method
US10500223B2 (en) * 2015-07-14 2019-12-10 Osaka University Therapeutic agent for myotonic dystrophy
US10888578B2 (en) 2016-12-19 2021-01-12 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
US11352624B2 (en) * 2013-09-04 2022-06-07 Cold Spring Harbor Laboratory Reducing nonsense-mediated MRNA decay

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KR102095478B1 (ko) 2010-05-28 2020-04-01 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
EP3489360A3 (fr) 2010-07-19 2019-08-28 Ionis Pharmaceuticals, Inc. Modulation de l'arn retenu nucléaire
US10017763B2 (en) 2010-09-03 2018-07-10 Sarepta Therapeutics, Inc. dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
EP2699269A1 (fr) * 2011-04-22 2014-02-26 Prosensa Technologies B.V. Nouveaux composés pour traiter, retarder et/ou prévenir un trouble génétique humain, tel que la dystrophie myotonique de type 1 (dm1)
EP2704749A1 (fr) 2011-05-05 2014-03-12 Sarepta Therapeutics, Inc. Conjugués peptides/oligonucléotides
WO2013033223A1 (fr) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Procédés et composés utiles dans des pathologies associées à des expansions répétées
CA2854907C (fr) 2011-11-18 2020-03-10 Sarepta Therapeutics, Inc. Oligonucleotides fonctionnellement modifies et sous-unites associees
MX358497B (es) 2012-03-20 2018-08-23 Sarepta Therapeutics Inc Conjugados de acido boronico de analogos de nucleotido.
US9909128B2 (en) * 2012-11-15 2018-03-06 The Regents Of The University Of California Splice modulating oligonucleotides that inhibit cancer
US10435430B2 (en) 2013-07-31 2019-10-08 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
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
EP3020403A1 (fr) 2014-11-14 2016-05-18 Universitat de Valéncia Composés pour le traitement de la dystrophie myotonique
EP3020404A1 (fr) 2014-11-14 2016-05-18 Universitat de Valéncia Caféine pour le traitement de la dystrophie myotonique de type 1 et de type 2
WO2017182581A1 (fr) * 2016-04-20 2017-10-26 Universität Basel Procédé d'évaluation d'un composé candidat (t) destiné au traitement de la dystrophie myotonique de type i (dm1)
ES2659845B1 (es) * 2016-09-19 2019-01-04 Univ Valencia Modulación de microRNAs contra la distrofia miotónica tipo 1 y antagonistas de microRNAs para ello
WO2023034870A2 (fr) 2021-09-01 2023-03-09 Ionis Pharmaceuticals, Inc. Composés et méthodes pour réduire l'expression de dmpk
WO2023168434A1 (fr) * 2022-03-04 2023-09-07 The Trustees Of Columbia University In The City Of New York Procédés et compositions pour réguler l'épissage de mapt pour la modélisation et le traitement de tauopathies

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JP2010504098A (ja) 2010-02-12
EP2560001A2 (fr) 2013-02-20
CA2981308C (fr) 2020-12-22
EP2066812A4 (fr) 2010-04-28
US9371527B2 (en) 2016-06-21
EP2066812A2 (fr) 2009-06-10
CA2664189C (fr) 2017-11-21
WO2008036406A2 (fr) 2008-03-27
EP2560001B1 (fr) 2016-04-13
JP5926475B2 (ja) 2016-05-25
EP3034083B1 (fr) 2020-12-09
US20150080452A1 (en) 2015-03-19
EP3034083A3 (fr) 2016-12-21
AU2007297535B2 (en) 2014-05-22
CA2981308A1 (fr) 2008-03-27
JP6259000B2 (ja) 2018-01-10
EP2560001A3 (fr) 2013-07-17
AU2007297535C1 (en) 2017-11-23
WO2008036406A3 (fr) 2009-04-30
EP3034083A2 (fr) 2016-06-22
CA2664189A1 (fr) 2008-03-27
JP2014055983A (ja) 2014-03-27
AU2007297535A1 (en) 2008-03-27
WO2008036406A9 (fr) 2008-07-03

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