IL141409A0 - Methods and compositions for use in spliceosome mediated rna trans-splicing - Google Patents

Methods and compositions for use in spliceosome mediated rna trans-splicing

Info

Publication number
IL141409A0
IL141409A0 IL14140999A IL14140999A IL141409A0 IL 141409 A0 IL141409 A0 IL 141409A0 IL 14140999 A IL14140999 A IL 14140999A IL 14140999 A IL14140999 A IL 14140999A IL 141409 A0 IL141409 A0 IL 141409A0
Authority
IL
Israel
Prior art keywords
splicing
compositions
methods
mediated rna
rna trans
Prior art date
Application number
IL14140999A
Other languages
English (en)
Original Assignee
Intronn Holdings Llc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority claimed from US09/133,717 external-priority patent/US6083702A/en
Application filed by Intronn Holdings Llc filed Critical Intronn Holdings Llc
Publication of IL141409A0 publication Critical patent/IL141409A0/xx

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    • CCHEMISTRY; METALLURGY
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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • C12N15/1027Mutagenizing nucleic acids by DNA shuffling, e.g. RSR, STEP, RPR
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C07K14/34Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria from Corynebacterium (G)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712Cystic fibrosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575Hormones
    • C07K14/59Follicle-stimulating hormone [FSH]; Chorionic gonadotropins, e.g.hCG [human chorionic gonadotropin]; Luteinising hormone [LH]; Thyroid-stimulating hormone [TSH]
    • CCHEMISTRY; METALLURGY
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/66General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/111Antisense spanning the whole gene, or a large part of it
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/44Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/44Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Toxicology (AREA)
  • Endocrinology (AREA)
  • Immunology (AREA)
  • Pulmonology (AREA)
  • Reproductive Health (AREA)
  • Epidemiology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
IL14140999A 1998-08-13 1999-08-12 Methods and compositions for use in spliceosome mediated rna trans-splicing IL141409A0 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US09/133,717 US6083702A (en) 1995-12-15 1998-08-13 Methods and compositions for use in spliceosome mediated RNA trans-splicing
US09/158,863 US6280978B1 (en) 1995-12-15 1998-09-23 Methods and compositions for use in spliceosome mediated RNA trans-splicing
PCT/US1999/018371 WO2000009734A2 (en) 1998-08-13 1999-08-12 Methods and compositions for use in spliceosome mediated rna trans-splicing

Publications (1)

Publication Number Publication Date
IL141409A0 true IL141409A0 (en) 2002-03-10

Family

ID=26831623

Family Applications (1)

Application Number Title Priority Date Filing Date
IL14140999A IL141409A0 (en) 1998-08-13 1999-08-12 Methods and compositions for use in spliceosome mediated rna trans-splicing

Country Status (7)

Country Link
US (1) US6280978B1 (enExample)
EP (2) EP1105513A2 (enExample)
JP (2) JP4353390B2 (enExample)
AU (1) AU773186B2 (enExample)
CA (1) CA2339300C (enExample)
IL (1) IL141409A0 (enExample)
WO (1) WO2000009734A2 (enExample)

Families Citing this family (52)

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US20030027250A1 (en) * 1995-12-15 2003-02-06 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20030077754A1 (en) * 1995-12-15 2003-04-24 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20060088938A1 (en) * 1995-12-15 2006-04-27 Mitchell Lloyd G Methods and compositions for use in spliceosome mediated RNA trans-splicing in plants
US20020193580A1 (en) * 1995-12-15 2002-12-19 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20020115207A1 (en) * 1995-12-15 2002-08-22 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
FR2790955B1 (fr) * 1999-03-19 2003-01-17 Assist Publ Hopitaux De Paris Utilisation d'oligonucleotides stabilises comme principe actif antitumoral
GB2362383B (en) * 2000-05-19 2003-12-31 Devgen Nv Gene expression system
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DE10057014B8 (de) * 2000-11-17 2008-04-30 Steven Dooley Verfahren zur Herstellung von zielzellspezifischen Arzneimitteln
EP1358203A4 (en) * 2001-01-08 2006-11-22 Intronn Inc METHOD AND COMPOSITION FOR CONNECTING IN SPLICEOSOME-MEDIATED RNA TRANS SPLICING
US20040126774A1 (en) * 2001-01-08 2004-07-01 Mitchell Lioyd G. Correction of factor VIII genetic defects using spliceosome mediated RNA trans splicing
DE10139492B4 (de) * 2001-08-13 2004-06-09 Eul, Joachim, Dr. Verfahren zur Reparatur einer mutierten RNA aus einer gendefekten DNA und zum gezielten Abtöten von Tumorzellen durch RNA-Transspleißen sowie Verfahren zum Nachweis von natürlich-transgespleißter zellulärer RNA
US20030153054A1 (en) * 2002-02-12 2003-08-14 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20050137153A1 (en) * 2002-02-20 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA)
EP1485397A4 (en) * 2002-02-25 2005-05-25 Intronn Inc IMAGING OF GENE EXPRESSION MEDIATED BY TRANS-SPISSAGE
US7399753B2 (en) * 2002-02-25 2008-07-15 Virxsys Corporation Trans-splicing mediated photodynamic therapy
US20040214263A1 (en) * 2002-03-20 2004-10-28 Mitchell Lloyd G. Spliceosome mediated RNA trans-splicing
US20040018520A1 (en) * 2002-04-22 2004-01-29 James Thompson Trans-splicing enzymatic nucleic acid mediated biopharmaceutical and protein
US20030204861A1 (en) * 2002-04-30 2003-10-30 Madaiah Puttaraju Transgenic animal model for spliceosome-mediated RNA trans-splicing
US7094399B2 (en) * 2002-05-08 2006-08-22 Intronn, Inc. Use of spliceosome mediated RNA trans-splicing to confer cell selective replication to adenoviruses
CA2488249A1 (en) * 2002-06-05 2003-12-18 Intronn, Inc. Spliceosome mediated rna trans-splicing in stem cells
EP1521766B1 (en) 2002-06-05 2012-11-07 VIRxSYS Corporation SPLICEOSOME MEDIATED RNA i TRANS /i -SPLICING AND CORRECTION OF FACTOR VIII GENETIC DEFECTS USING SPLICEOSOME MEDIATED RNA TRANS SPLING
US20040018622A1 (en) * 2002-07-17 2004-01-29 Mitchell Lloyd G. Spliceosome mediated RNA trans-splicing for correction of skin disorders
DE60333032D1 (de) * 2002-10-23 2010-07-29 Virxsys Corp Screening-verfahren zur identifizierung wirksamer prä-trans-spleissmoleküle
AU2003226642A1 (en) * 2003-03-31 2004-10-25 Council Of Scientific And Industrial Research Stn gene oligonucleotide primers for detecting salmonella species and detection prosses using the same
US7968334B2 (en) * 2004-01-23 2011-06-28 Virxsys Corporation Expression of apoAI and variants thereof using spliceosome mediated RNA trans-splicing
WO2005070948A1 (en) * 2004-01-23 2005-08-04 Intronn, Inc. Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated rna trans splicing
US20060177933A1 (en) * 2004-01-23 2006-08-10 Madaiah Puttaraju Expression of apoA-1 and variants thereof using spliceosome mediated RNA trans-splicing
US20060094110A1 (en) * 2004-07-30 2006-05-04 Mcgarrity Gerard J Use of spliceosome mediated RNA trans-splicing for immunotherapy
US20060134658A1 (en) * 2004-08-09 2006-06-22 Garcia-Blanco Mariano A Use of RNA trans-splicing for generation of interfering RNA molecules
DE102004047492B4 (de) * 2004-09-23 2006-07-20 Jost-Werke Gmbh & Co. Kg Verfahren zum Übertragen von elektrischer, pneumatischer oder hydraulischer Energie sowie ein Energieübertragungssystem
EP1797184B1 (en) * 2004-10-08 2012-09-26 VIRxSYS Corporation Targeted trans-splicing of highly abundant transcripts for in vivo production of recombinant proteins
US7871795B2 (en) * 2004-10-08 2011-01-18 Virxsys Corporation Targeted trans-splicing of highly abundant transcripts for in vivo production of recombinant proteins
US7879321B2 (en) * 2004-10-08 2011-02-01 Virxsys Corporation Use of RNA trans-splicing for antibody gene transfer and antibody polypeptide production
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EP2151248A1 (en) 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
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WO2013016595A1 (en) 2011-07-28 2013-01-31 The Regents Of The University Of California Exonic splicing enhancers and exonic splicing silencers
EP2898072A1 (en) 2012-09-24 2015-07-29 Yissum Research Development Company of the Hebrew University of Jerusalem Ltd. Restoration of the cftr function by splicing modulation
CA2879426A1 (en) * 2013-01-14 2014-07-17 Cellecta, Inc. Methods and compositions for single cell expression profiling
WO2017087900A1 (en) 2015-11-19 2017-05-26 The Trustees Of The University Of Pennsylvania Compositions and methods for correction of heritable ocular disease
CN109642241B (zh) * 2016-04-01 2023-06-27 新加坡国立大学 反式剪接RNA(tsRNA)
KR102866133B1 (ko) 2018-04-17 2025-09-30 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 트랜스-스플라이싱 분자
KR20220044811A (ko) 2019-08-16 2022-04-11 메사추세츠 인스티튜트 오브 테크놀로지 Crispr/cas13을 사용하는 표적화된 트랜스-이어맞추기
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US12351818B2 (en) 2022-11-23 2025-07-08 Amber Bio Inc. Gene-modifying endonucleases
WO2025128343A1 (en) 2023-12-11 2025-06-19 Just-Evotec Biologics, Inc. Protein expression using trans-splicing and split selectable markers

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US5498531A (en) * 1993-09-10 1996-03-12 President And Fellows Of Harvard College Intron-mediated recombinant techniques and reagents
US5667969A (en) * 1993-11-12 1997-09-16 University Research Corporation Alteration of sequence of a deleterious target molecule by ribozyme catalyzed trans-splicing
EP0883344B2 (en) * 1995-12-15 2010-06-09 VIRxSYS Corporation Therapeutic molecules generated by trans-splicing

Also Published As

Publication number Publication date
US6280978B1 (en) 2001-08-28
JP2004504001A (ja) 2004-02-12
AU5673599A (en) 2000-03-06
EP1105513A2 (en) 2001-06-13
CA2339300A1 (en) 2000-02-24
WO2000009734A9 (en) 2000-07-13
WO2000009734A3 (en) 2000-05-18
AU773186B2 (en) 2004-05-20
EP1489185A1 (en) 2004-12-22
CA2339300C (en) 2015-11-24
JP4353390B2 (ja) 2009-10-28
WO2000009734A2 (en) 2000-02-24
JP2007312780A (ja) 2007-12-06

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