SI1496944T1 - Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov - Google Patents
Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinovInfo
- Publication number
- SI1496944T1 SI1496944T1 SI200331424T SI200331424T SI1496944T1 SI 1496944 T1 SI1496944 T1 SI 1496944T1 SI 200331424 T SI200331424 T SI 200331424T SI 200331424 T SI200331424 T SI 200331424T SI 1496944 T1 SI1496944 T1 SI 1496944T1
- Authority
- SI
- Slovenia
- Prior art keywords
- vectors
- expression systems
- raav
- genetic modification
- methods
- Prior art date
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2750/14145—Special targeting system for viral vectors
-
- 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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
-
- 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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
- C12N2810/80—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
- C12N2810/85—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
- C12N2810/858—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian from apolipopeptides
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Virology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US37731502P | 2002-05-01 | 2002-05-01 | |
EP03786493A EP1496944B1 (en) | 2002-05-01 | 2003-05-01 | Improved raav expression systems for genetic modification of specific capsid proteins |
PCT/US2003/013583 WO2004027019A2 (en) | 2002-05-01 | 2003-05-01 | Improved raav expression systems for genetic modification of specific capsid proteins |
Publications (1)
Publication Number | Publication Date |
---|---|
SI1496944T1 true SI1496944T1 (sl) | 2009-02-28 |
Family
ID=32030574
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SI200331424T SI1496944T1 (sl) | 2002-05-01 | 2003-05-01 | Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov |
Country Status (11)
Country | Link |
---|---|
US (2) | US8802080B2 (sl) |
EP (1) | EP1496944B1 (sl) |
AT (1) | ATE405295T1 (sl) |
AU (1) | AU2003295312B2 (sl) |
CA (1) | CA2483624A1 (sl) |
DE (1) | DE60323078D1 (sl) |
DK (1) | DK1496944T3 (sl) |
HK (1) | HK1071518A1 (sl) |
NZ (2) | NZ561656A (sl) |
SI (1) | SI1496944T1 (sl) |
WO (1) | WO2004027019A2 (sl) |
Families Citing this family (75)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20060093589A1 (en) * | 2004-02-19 | 2006-05-04 | Warrington Kenneth H | Vp2-modified raav vector compositions and uses therefor |
AU2004226961B2 (en) * | 2002-05-01 | 2009-06-11 | University Of Florida Research Foundation, Inc. | VP2-modified rAAV vector compositions and uses therefor |
SI1496944T1 (sl) | 2002-05-01 | 2009-02-28 | Univ Florida | Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov |
US20040179028A1 (en) * | 2003-03-12 | 2004-09-16 | Fuji Photo Film Co., Ltd. | Pixel defect correcting method, color mura correcting method and image display device |
EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
ES2329636B2 (es) | 2006-02-17 | 2010-07-26 | Universitat De Valencia, Estudi General (Participa Con El 70%) | Uso del factor pedf para inducir la auto-renovacion de celulas madre. |
DK2191001T3 (en) | 2007-04-09 | 2016-09-19 | Univ Florida | RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF |
US9611302B2 (en) | 2007-04-09 | 2017-04-04 | University Of Florida Research Foundation, Inc. | High-transduction-efficiency RAAV vectors, compositions, and methods of use |
US9725485B2 (en) * | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
WO2009105669A2 (en) * | 2008-02-20 | 2009-08-27 | Genzyme Corporation | Angiogenesis inhibition |
ES2538468T3 (es) | 2008-05-20 | 2015-06-22 | Eos Neuroscience, Inc. | Vectores para la administración de proteínas sensibles a la luz y métodos para su utilización |
US8889641B2 (en) | 2009-02-11 | 2014-11-18 | The University Of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
JP2012519008A (ja) | 2009-03-04 | 2012-08-23 | ドイチェス クレブスフォルシュングスツェントルム | 集合活性化タンパク質(aap)およびvp3から本質的になるパルボウイルス粒子を製造するためのaapの使用 |
US9884909B2 (en) | 2010-12-01 | 2018-02-06 | Alderbio Holdings Llc | Anti-NGF compositions and use thereof |
US9067988B2 (en) | 2010-12-01 | 2015-06-30 | Alderbio Holdings Llc | Methods of preventing or treating pain using anti-NGF antibodies |
US9539324B2 (en) | 2010-12-01 | 2017-01-10 | Alderbio Holdings, Llc | Methods of preventing inflammation and treating pain using anti-NGF compositions |
US9078878B2 (en) | 2010-12-01 | 2015-07-14 | Alderbio Holdings Llc | Anti-NGF antibodies that selectively inhibit the association of NGF with TrkA, without affecting the association of NGF with p75 |
US11214610B2 (en) | 2010-12-01 | 2022-01-04 | H. Lundbeck A/S | High-purity production of multi-subunit proteins such as antibodies in transformed microbes such as Pichia pastoris |
CN103619879A (zh) | 2010-12-01 | 2014-03-05 | 奥尔德生物控股有限责任公司 | 抗ngf组合物及其用途 |
EP2500434A1 (en) * | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery |
CA2856137A1 (en) | 2011-11-22 | 2013-05-30 | The Children's Hospital Of Philadelphia | Virus vectors for highly efficient transgene delivery |
US10294281B2 (en) | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
DK3024498T3 (da) | 2013-07-22 | 2020-03-02 | Childrens Hospital Philadelphia | Aav-variant og sammensætninger, fremgangsmåder og anvendelser til genoverførsel til celler, organer og væv |
CN104592364B (zh) * | 2013-10-30 | 2018-05-01 | 北京大学 | 定点突变和定点修饰的腺相关病毒、其制备方法及应用 |
CN115430458A (zh) * | 2013-12-06 | 2022-12-06 | 庄信万丰股份有限公司 | 冷启动催化剂和其在排气系统中的用途 |
GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
US10308957B2 (en) | 2014-03-04 | 2019-06-04 | University Of Florida Research Foundation, Inc. | rAAV vectors and methods for transduction of photoreceptors and RPE cells |
CN114231563A (zh) | 2014-05-02 | 2022-03-25 | 建新公司 | 用于视网膜及cns基因疗法的aav载体 |
WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
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US10597660B2 (en) | 2014-11-14 | 2020-03-24 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
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US10799566B2 (en) | 2015-06-23 | 2020-10-13 | The Children's Hospital Of Philadelphia | Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues |
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KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
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AU2017312951B2 (en) | 2016-08-15 | 2024-02-08 | Genzyme Corporation | Methods for detecting AAV |
CN110650673B (zh) | 2016-08-30 | 2024-04-09 | 加利福尼亚大学董事会 | 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统 |
US11192925B2 (en) | 2016-10-19 | 2021-12-07 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
KR20190117571A (ko) | 2017-02-15 | 2019-10-16 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 혈관계를 통한 유전자 전달 방법 및 조성물 |
CN110392607A (zh) * | 2017-03-13 | 2019-10-29 | 三菱化学株式会社 | 过渡金属负载沸石和其制造方法以及氮氧化物净化用催化剂和其使用方法 |
US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
CN117801075A (zh) * | 2017-03-15 | 2024-04-02 | 北卡罗来纳-查佩尔山大学 | 多倍体腺相关病毒载体及其制备和使用方法 |
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AU2018352236A1 (en) | 2017-10-16 | 2020-04-23 | The Curators Of The University Of Missouri | Treatment of amyotrophic lateral sclerosis (ALS) |
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US20210107947A1 (en) * | 2018-02-15 | 2021-04-15 | Tomas Björklund | Modified viral capsids |
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CN117535247A (zh) * | 2018-05-07 | 2024-02-09 | 北卡罗来纳-查佩尔山大学 | 合理的多倍体腺相关病毒载体及其制造和使用方法 |
US11821009B2 (en) | 2018-05-15 | 2023-11-21 | Cornell University | Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery |
US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
US20210317478A1 (en) * | 2018-08-21 | 2021-10-14 | Massachusetts Eye And Ear Infirmary | Compositions and methods for modulating transduction efficiency of adeno-associated viruses |
AU2020229085A1 (en) | 2019-02-25 | 2021-08-19 | Friedrich Miescher Institute For Biomedical Research | Compositions and methods to treat bietti crystalline dystrophy |
CN113474461A (zh) | 2019-02-25 | 2021-10-01 | 诺华股份有限公司 | 治疗bietti晶体营养不良的组合物和方法 |
TW202102525A (zh) | 2019-03-21 | 2021-01-16 | 美商史崔德生物公司 | 重組腺相關病毒載體 |
US20210082541A1 (en) * | 2019-08-31 | 2021-03-18 | Wyatt Technology Corporation | Measuring attributes of a viral gene delivery vehicle sample via separation |
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WO2024006433A1 (en) * | 2022-06-30 | 2024-01-04 | Carnegie Mellon University | Method for production of virus-like particles for gene delivery |
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WO1994029469A2 (en) * | 1993-06-07 | 1994-12-22 | Vical Incorporated | Plasmids suitable for gene therapy |
US6204059B1 (en) * | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
US5741683A (en) * | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
JP2001500376A (ja) * | 1996-09-06 | 2001-01-16 | カイロン コーポレイション | 組換えaavベクターを使用する治療分子の肝臓特異的送達のための方法および組成物 |
US6221646B1 (en) * | 1997-07-31 | 2001-04-24 | Chiron Corporation | Materials and methods for simplified AAV production |
US6943153B1 (en) * | 1999-03-15 | 2005-09-13 | The Regents Of The University Of California | Use of recombinant gene delivery vectors for treating or preventing diseases of the eye |
CA2367375A1 (en) | 1999-03-15 | 2000-09-21 | Chiron Corporation | Use of recombinant gene delivery vectors for treating or preventing diseases of the eye |
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WO2002024234A2 (en) | 2000-09-20 | 2002-03-28 | The Regents Of The University Of California | Use of recombinant gene delivery vectors for treating or preventing diseases of the eye |
AU2002248297A1 (en) * | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
US6692815B2 (en) * | 2001-06-15 | 2004-02-17 | Vinylex Corporation | Extruded plastic lumber and method of manufacture |
US6723551B2 (en) * | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
AU2003221733A1 (en) * | 2002-04-17 | 2003-11-03 | University Of Florida Research Foundation, Inc. | Improved raav vectors |
SI1496944T1 (sl) | 2002-05-01 | 2009-02-28 | Univ Florida | Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov |
AU2004226961B2 (en) | 2002-05-01 | 2009-06-11 | University Of Florida Research Foundation, Inc. | VP2-modified rAAV vector compositions and uses therefor |
WO2004020600A2 (en) * | 2002-08-28 | 2004-03-11 | University Of Florida | Modified aav |
-
2003
- 2003-05-01 SI SI200331424T patent/SI1496944T1/sl unknown
- 2003-05-01 DE DE60323078T patent/DE60323078D1/de not_active Expired - Fee Related
- 2003-05-01 CA CA002483624A patent/CA2483624A1/en not_active Abandoned
- 2003-05-01 NZ NZ561656A patent/NZ561656A/en unknown
- 2003-05-01 AU AU2003295312A patent/AU2003295312B2/en not_active Ceased
- 2003-05-01 WO PCT/US2003/013583 patent/WO2004027019A2/en not_active Application Discontinuation
- 2003-05-01 AT AT03786493T patent/ATE405295T1/de not_active IP Right Cessation
- 2003-05-01 NZ NZ536274A patent/NZ536274A/en unknown
- 2003-05-01 US US10/513,059 patent/US8802080B2/en active Active
- 2003-05-01 EP EP03786493A patent/EP1496944B1/en not_active Expired - Lifetime
- 2003-05-01 DK DK03786493T patent/DK1496944T3/da active
-
2005
- 2005-05-24 HK HK05104362.7A patent/HK1071518A1/xx not_active IP Right Cessation
-
2014
- 2014-07-08 US US14/326,293 patent/US20150005369A1/en not_active Abandoned
Also Published As
Publication number | Publication date |
---|---|
EP1496944B1 (en) | 2008-08-20 |
ATE405295T1 (de) | 2008-09-15 |
US20150005369A1 (en) | 2015-01-01 |
DK1496944T3 (da) | 2008-12-01 |
AU2003295312A1 (en) | 2004-04-08 |
CA2483624A1 (en) | 2004-04-01 |
EP1496944A4 (en) | 2006-03-22 |
US8802080B2 (en) | 2014-08-12 |
NZ561656A (en) | 2009-03-31 |
DE60323078D1 (de) | 2008-10-02 |
WO2004027019A3 (en) | 2004-08-05 |
US20060088936A1 (en) | 2006-04-27 |
WO2004027019A2 (en) | 2004-04-01 |
NZ536274A (en) | 2008-04-30 |
AU2003295312B2 (en) | 2008-08-14 |
HK1071518A1 (en) | 2005-07-22 |
EP1496944A2 (en) | 2005-01-19 |
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