CA2564092A1 - Methods and compositions for the treatment of polycystic diseases - Google Patents
Methods and compositions for the treatment of polycystic diseases Download PDFInfo
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- CA2564092A1 CA2564092A1 CA002564092A CA2564092A CA2564092A1 CA 2564092 A1 CA2564092 A1 CA 2564092A1 CA 002564092 A CA002564092 A CA 002564092A CA 2564092 A CA2564092 A CA 2564092A CA 2564092 A1 CA2564092 A1 CA 2564092A1
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- antibody
- agent
- polynucleotide
- gene
- molecule
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- 238000000034 method Methods 0.000 title claims abstract description 34
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title claims abstract description 9
- 201000010099 disease Diseases 0.000 title abstract description 6
- 239000000203 mixture Substances 0.000 title abstract description 5
- 108090000623 proteins and genes Proteins 0.000 claims abstract 17
- 230000014509 gene expression Effects 0.000 claims abstract 14
- 210000001519 tissue Anatomy 0.000 claims abstract 7
- 230000002401 inhibitory effect Effects 0.000 claims abstract 6
- 230000004071 biological effect Effects 0.000 claims abstract 5
- 230000015572 biosynthetic process Effects 0.000 claims abstract 3
- 230000003902 lesion Effects 0.000 claims abstract 3
- 208000010061 Autosomal Dominant Polycystic Kidney Diseases 0.000 claims description 5
- 208000022185 autosomal dominant polycystic kidney disease Diseases 0.000 claims description 5
- 230000002440 hepatic effect Effects 0.000 claims description 2
- 239000003795 chemical substances by application Substances 0.000 claims 20
- 108091033319 polynucleotide Proteins 0.000 claims 19
- 102000040430 polynucleotide Human genes 0.000 claims 19
- 239000002157 polynucleotide Substances 0.000 claims 19
- 150000003384 small molecules Chemical group 0.000 claims 5
- 108090000994 Catalytic RNA Proteins 0.000 claims 3
- 102000053642 Catalytic RNA Human genes 0.000 claims 3
- 102000008394 Immunoglobulin Fragments Human genes 0.000 claims 3
- 108010021625 Immunoglobulin Fragments Proteins 0.000 claims 3
- 108091008103 RNA aptamers Proteins 0.000 claims 3
- 230000004913 activation Effects 0.000 claims 3
- 230000000692 anti-sense effect Effects 0.000 claims 3
- 230000000694 effects Effects 0.000 claims 3
- 230000004481 post-translational protein modification Effects 0.000 claims 3
- 239000002243 precursor Substances 0.000 claims 3
- 108091092562 ribozyme Proteins 0.000 claims 3
- 102100031168 CCN family member 2 Human genes 0.000 claims 2
- 101000777550 Homo sapiens CCN family member 2 Proteins 0.000 claims 2
- 230000005856 abnormality Effects 0.000 claims 2
- 239000003446 ligand Substances 0.000 claims 2
- 210000004923 pancreatic tissue Anatomy 0.000 claims 1
- 210000005084 renal tissue Anatomy 0.000 claims 1
- 230000037416 cystogenesis Effects 0.000 abstract description 2
- 102000015225 Connective Tissue Growth Factor Human genes 0.000 abstract 2
- 108010039419 Connective Tissue Growth Factor Proteins 0.000 abstract 2
- 208000035475 disorder Diseases 0.000 abstract 2
- 230000002159 abnormal effect Effects 0.000 abstract 1
- 230000003190 augmentative effect Effects 0.000 abstract 1
- 230000002596 correlated effect Effects 0.000 abstract 1
- 208000031513 cyst Diseases 0.000 abstract 1
- 230000001575 pathological effect Effects 0.000 abstract 1
- 206010019646 Hepatic cyst Diseases 0.000 description 1
- 208000000407 Pancreatic Cyst Diseases 0.000 description 1
- 238000003745 diagnosis Methods 0.000 description 1
- 230000002526 effect on cardiovascular system Effects 0.000 description 1
- 230000002068 genetic effect Effects 0.000 description 1
- 210000003734 kidney Anatomy 0.000 description 1
- 208000017169 kidney disease Diseases 0.000 description 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/22—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
-
- 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
- A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
-
- 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
- A61P15/00—Drugs for genital or sexual disorders; Contraceptives
- A61P15/08—Drugs for genital or sexual disorders; Contraceptives for gonadal disorders or for enhancing fertility, e.g. inducers of ovulation or of spermatogenesis
-
- 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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/12—Drugs for disorders of the metabolism for electrolyte homeostasis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- 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
-
- 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
- A61P9/12—Antihypertensives
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Reproductive Health (AREA)
- Urology & Nephrology (AREA)
- Diabetes (AREA)
- Genetics & Genomics (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Pain & Pain Management (AREA)
- Rheumatology (AREA)
- Gastroenterology & Hepatology (AREA)
- Neurology (AREA)
- Immunology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Neurosurgery (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gynecology & Obstetrics (AREA)
- Pregnancy & Childbirth (AREA)
- Biomedical Technology (AREA)
- Endocrinology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
Abstract
This invention provides compositions and methods to diagnose and treat polycystic disorders by inhibiting the biological activity of a gene now correlated with appearance of this disorder. By way of illustrative only, the Connective Tissue Growth Factor (CTGF) gene is an example of such a gene. Also provided by this invention are compositions and methods to treat or ameliorate abnormal cystic lesions and diseases associated with the formation of cysts in tissue. The methods and compositions treat and ameliorate pathological cyst formation in tissue by inhibiting or augmenting gene expression or the biological activity of its gene expression product or its receptor.
Description
DEMANDE OU BREVET VOLUMINEUX
LA PRESENTE PARTIE DE CETTE DEMANDE OU CE BREVET COMPREND
PLUS D'UN TOME.
NOTE : Pour les tomes additionels, veuillez contacter le Bureau canadien des brevets JUMBO APPLICATIONS/PATENTS
THIS SECTION OF THE APPLICATION/PATENT CONTAINS MORE THAN ONE
VOLUME
NOTE: For additional volumes, please contact the Canadian Patent Office NOM DU FICHIER / FILE NAME:
NOTE POUR LE TOME / VOLUME NOTE:
METHODS AND COMPOSITIONS FOR THE TREATMENT OF
POLYCYSTIC DISEASES
CROSS-REFERENCE TO RELATED APPLICATIONS
This application claims priority under 35 U.S.C. 119(e) to U.S.
Provisional Applications, U.S. Serial Nos. 60/566,670 and 60/590,385, filed April 29, 2004 and July 22, 2004, respectively. The contents of these applications are incorporated by reference into the present disclosure.
TECHNICAL FIELD OF THE INVENTION
This invention is related to the area of polycystic diseases and to the diagnosis and treatment of such diseases.
BACKGROUND OF THE INVENTION
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common genetic renal disorder occurring in 1:1000 individuals and is characterized by focal cyst formation in all tubular segments (Friedman, J.
Cystic Diseases of the Kidney, in PRINCIPLES AND PRACTICE OF MEDICAL
GENETICS (A. Emery and D. Rimoin, Eds.) pp. 1002-1010, Churchill Livingston, Edinburgh, U.K. (1983); Striker & Striker (1986) Am. J. Nephrol.
6:161-164. Extrarenal manifestations include hepatic and pancreatic cysts as well as cardiovascular complications. Gabow & Grantham (1997)
LA PRESENTE PARTIE DE CETTE DEMANDE OU CE BREVET COMPREND
PLUS D'UN TOME.
NOTE : Pour les tomes additionels, veuillez contacter le Bureau canadien des brevets JUMBO APPLICATIONS/PATENTS
THIS SECTION OF THE APPLICATION/PATENT CONTAINS MORE THAN ONE
VOLUME
NOTE: For additional volumes, please contact the Canadian Patent Office NOM DU FICHIER / FILE NAME:
NOTE POUR LE TOME / VOLUME NOTE:
METHODS AND COMPOSITIONS FOR THE TREATMENT OF
POLYCYSTIC DISEASES
CROSS-REFERENCE TO RELATED APPLICATIONS
This application claims priority under 35 U.S.C. 119(e) to U.S.
Provisional Applications, U.S. Serial Nos. 60/566,670 and 60/590,385, filed April 29, 2004 and July 22, 2004, respectively. The contents of these applications are incorporated by reference into the present disclosure.
TECHNICAL FIELD OF THE INVENTION
This invention is related to the area of polycystic diseases and to the diagnosis and treatment of such diseases.
BACKGROUND OF THE INVENTION
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common genetic renal disorder occurring in 1:1000 individuals and is characterized by focal cyst formation in all tubular segments (Friedman, J.
Cystic Diseases of the Kidney, in PRINCIPLES AND PRACTICE OF MEDICAL
GENETICS (A. Emery and D. Rimoin, Eds.) pp. 1002-1010, Churchill Livingston, Edinburgh, U.K. (1983); Striker & Striker (1986) Am. J. Nephrol.
6:161-164. Extrarenal manifestations include hepatic and pancreatic cysts as well as cardiovascular complications. Gabow & Grantham (1997)
Claims (29)
1. A method for inhibiting cystic disorders or abnormalities in a suitable tissue by contacting the tissue with an effective amount of an agent that modulates the biological activity of a gene or polynucleotide identified in Tables 2 through 5, thereby inhibiting cystic abnormalities.
2. The method of claim 1, wherein the suitable tissue is selected from the group consisting of tubular kidney tissue, hepatic tissue and pancreatic tissue.
3. The method of claim 2, wherein the suitable tissue is isolated from a subject suffering from ADPKD.
4. The method of claim 1, wherein the modulating agent is a polynucleotide that modulates the activity or expression of a polynucleotide or gene identified in Tables 2 through 5.
5. The method of claim 4, wherein the agent is an antibody or ligand that specifically binds to the expression product of the gene or polynucleotide.
6. The method of claim 4, wherein the agent is selected from the group consisting of an antisense polynucleotide, a ribozyme and a multivalent RNA aptamer.
7. The method of claim 5, wherein the agent is selected from the group consisting of an antibody, an antibody derivative, and an antibody variant.
8. The method of claim 5, wherein the agent is a polyclonal antibody or a monoclonal antibody.
9. The method of claim 7, wherein the agent is selected from the group consisting of an antibody fragment, a humanized antibody and a chimeric antibody.
10. The method of claim 1, wherein the agent is a small molecule that modifies, blocks or augments post-translational modification the expression product of the gene or polynucleotide.
11. The method of claim 1, wherein the agent is a small molecule that modulates the activation of a precursor of the expression product of the polynucleotide or gene.
12. A method for inhibiting the formation of polycystic lesions in a subject, comprising delivering to the subject an effective amount of an agent that modulates the biological activity of a gene identified in Tables 2 through 5, thereby inhibiting the formation of polycystic lesions.
13. The method of claim 12, wherein the modulating agent is a polynucleotide that inhibits or augments the activity or expression of a CTGF polynucleotide.
14. The method of claim 12, wherein the agent is an antibody or ligand that specifically binds to the expression product of the gene or the polynucleotide.
15. The method of claim 13, wherein the agent is selected from the group consisting of an antisense polynucleotide, a ribozyme and a multivalent RNA aptamer.
16. The method of claim 14, wherein the agent is an antibody selected from the group consisting of a monoclonal antibody, an antibody derivative and an antibody variant.
17. The method of claim 14, wherein the agent is a polyclonal antibody or a monoclonal antibody.
18. The method of claim 17, wherein the agent is selected from the group consisting of an antibody fragment, a humanized antibody and a chimeric antibody.
19. The method of claim 12, wherein the agent is a small molecule that modifies, blocks, or augments post-translational modification of a polynucleotide or gene identified in Tables 2 through 5.
20. The method of claim 12, wherein the agent is a molecule that modifies the activation of a precursor of the expression product of the polynucleotide or gene identified in Tables 2 through 5.
21. A method for preventing or treating Autosomal Dominant Polycystic Kidney Disease (ADPKD) in a suitable subject, comprising delivering an effective amount of an isolated molecule that modulates polycystic biological activity of a gene or its expression product identified in Tables 2 through 5, to a subject in need thereof.
22. The method of claim 21, wherein the isolated molecule is a polynucleotide that modulates the activity or expression of a CTGF
polynucleotide.
polynucleotide.
23. The method of claim 21, wherein the molecule is an antibody that specifically binds to the expression product of the gene or the polynucleotide.
24. The method of claim 22, wherein the molecule is selected from the group consisting of an antisense polynucleotide, a small molecule, a ribozyme, a multivalent RNA aptamer.
25. The method of claim 22, wherein the molecule is an antibody selected from the group consisting of an antibody, an antibody derivative and an antibody variant.
26. The method of claim 22, wherein the agent is a polyclonal antibody or a monoclonal antibody.
27. The method of claim 26, wherein the agent is selected from the group consisting of an antibody fragment, a humanized antibody and a chimeric antibody.
28. The method of claim 22, wherein the molecule is a small molecule that modifies, inhibits or augments post-translational modification of an expression product of a gene or a polynucleotide or gene identified in Tables 2 through 5.
29. The method of claim 22, wherein the molecule is a molecule that modifies the activation of a precursor of the expression product of a polynucleotide or gene identified in Tables 2 through 5.
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US56667004P | 2004-04-29 | 2004-04-29 | |
| US60/566,670 | 2004-04-29 | ||
| US59038504P | 2004-07-22 | 2004-07-22 | |
| US60/590,385 | 2004-07-22 | ||
| PCT/US2005/014982 WO2005117941A2 (en) | 2004-04-29 | 2005-04-29 | Methods and compositions for the treatment of polycystic diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2564092A1 true CA2564092A1 (en) | 2005-12-15 |
Family
ID=35463346
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA002564092A Abandoned CA2564092A1 (en) | 2004-04-29 | 2005-04-29 | Methods and compositions for the treatment of polycystic diseases |
Country Status (6)
| Country | Link |
|---|---|
| EP (1) | EP1740221A4 (en) |
| JP (1) | JP2007535562A (en) |
| BR (1) | BRPI0510367A (en) |
| CA (1) | CA2564092A1 (en) |
| MX (1) | MXPA06012446A (en) |
| WO (1) | WO2005117941A2 (en) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG177194A1 (en) | 2005-12-08 | 2012-01-30 | Medarex Inc | Human monoclonal antibodies to protein tyrosine kinase 7 (ptk7) and methods for using anti-ptk7 antibodies |
| EP1808694A1 (en) * | 2006-01-17 | 2007-07-18 | Universitätsklinikum Freiburg | Method for diagnosing polycystic kidney disease |
| DK2076268T3 (en) * | 2006-10-19 | 2013-04-22 | Genzyme Corp | Roscovitine for the treatment of certain cystic diseases |
| NZ591416A (en) | 2008-08-25 | 2012-11-30 | Excaliard Pharmaceuticals Inc | Antisense oligonucleotides directed against connective tissue growth factor and uses thereof |
| US8946172B2 (en) | 2008-08-25 | 2015-02-03 | Excaliard Pharmaceuticals, Inc. | Method for reducing scarring during wound healing using antisense compounds directed to CTGF |
| PL2670411T3 (en) | 2011-02-02 | 2019-09-30 | Excaliard Pharmaceuticals, Inc. | Antisense compounds targeting connective tissue growth factor (ctgf) for use in a method of treating keloids or hypertrophic scars |
| JP5995207B2 (en) * | 2014-11-28 | 2016-09-21 | 学校法人 学習院 | Boron-containing porphyrin derivatives |
| KR102288447B1 (en) * | 2018-12-28 | 2021-08-10 | 서울대학교산학협력단 | Composition for diagnosing polycystic kidney disease comprising agent for detecting CTGF and use thereof |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5789189A (en) * | 1993-09-24 | 1998-08-04 | The Regents Of The University Of California | Inhibition of cyst formation by cytoskeletal specific drugs |
| US6562618B1 (en) * | 1997-12-25 | 2003-05-13 | Japan Tobacco, Inc. | Monoclonal antibody against connective tissue growth factor and medicinal uses thereof |
| US6875747B1 (en) * | 1999-05-24 | 2005-04-05 | Avi Bio Pharma, Inc. | Antisense to c-myc for treatment of polycystic kidney disease |
| US20030113816A1 (en) * | 2001-09-18 | 2003-06-19 | Weitz Stephen L. | Methods of assaying connective tissue growth factor |
| US6965025B2 (en) * | 2001-12-10 | 2005-11-15 | Isis Pharmaceuticals, Inc. | Antisense modulation of connective tissue growth factor expression |
-
2005
- 2005-04-29 JP JP2007511046A patent/JP2007535562A/en not_active Withdrawn
- 2005-04-29 MX MXPA06012446A patent/MXPA06012446A/en not_active Application Discontinuation
- 2005-04-29 WO PCT/US2005/014982 patent/WO2005117941A2/en active Application Filing
- 2005-04-29 BR BRPI0510367-3A patent/BRPI0510367A/en not_active IP Right Cessation
- 2005-04-29 CA CA002564092A patent/CA2564092A1/en not_active Abandoned
- 2005-04-29 EP EP05804799A patent/EP1740221A4/en not_active Withdrawn
Also Published As
| Publication number | Publication date |
|---|---|
| EP1740221A4 (en) | 2009-01-07 |
| EP1740221A2 (en) | 2007-01-10 |
| WO2005117941A2 (en) | 2005-12-15 |
| WO2005117941A3 (en) | 2006-05-18 |
| MXPA06012446A (en) | 2007-01-17 |
| JP2007535562A (en) | 2007-12-06 |
| BRPI0510367A (en) | 2007-11-06 |
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