CA2339300C - Methods and compositions for use in spliceosome mediated rna trans-splicing - Google Patents

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

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Publication number
CA2339300C
CA2339300C CA2339300A CA2339300A CA2339300C CA 2339300 C CA2339300 C CA 2339300C CA 2339300 A CA2339300 A CA 2339300A CA 2339300 A CA2339300 A CA 2339300A CA 2339300 C CA2339300 C CA 2339300C
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trans
target
nucleic acid
acid molecule
mrna
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Expired - Fee Related
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CA2339300A
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English (en)
French (fr)
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CA2339300A1 (en
Inventor
Lloyd G. Mitchell
Mariano A. Garcia-Blanco
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VirxSys Corp
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VirxSys Corp
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Priority claimed from US09/133,717 external-priority patent/US6083702A/en
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Publication of CA2339300A1 publication Critical patent/CA2339300A1/en
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Publication of CA2339300C publication Critical patent/CA2339300C/en
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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
    • 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
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    • 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)
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    • 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
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    • 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]
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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
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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
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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
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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
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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
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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
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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
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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
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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
    • 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)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Toxicology (AREA)
  • Endocrinology (AREA)
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  • Immunology (AREA)
  • Epidemiology (AREA)
  • Reproductive Health (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA2339300A 1998-08-13 1999-08-12 Methods and compositions for use in spliceosome mediated rna trans-splicing Expired - Fee Related CA2339300C (en)

Applications Claiming Priority (5)

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
US09/133,717 1998-08-13
US09/158,863 1998-09-23
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 (2)

Publication Number Publication Date
CA2339300A1 CA2339300A1 (en) 2000-02-24
CA2339300C true CA2339300C (en) 2015-11-24

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CA2339300A Expired - Fee Related CA2339300C (en) 1998-08-13 1999-08-12 Methods and compositions for use in spliceosome mediated rna trans-splicing

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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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020115207A1 (en) * 1995-12-15 2002-08-22 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20020193580A1 (en) * 1995-12-15 2002-12-19 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20030027250A1 (en) * 1995-12-15 2003-02-06 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
US20030077754A1 (en) * 1995-12-15 2003-04-24 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
WO2002008408A2 (en) * 2000-07-21 2002-01-31 Trustees Of Boston University Modular vector systems
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)
CA2477295A1 (en) * 2002-02-25 2003-09-04 Intronn, Inc. Trans-splicing mediated imaging of gene expression
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
JP2005536231A (ja) * 2002-05-08 2005-12-02 イントロン,インコーポレーテッド アデノウイルスに細胞選択的複製をもたらすための、スプライセオソーム媒介型rnaトランススプライシングの使用
WO2003104412A2 (en) 2002-06-05 2003-12-18 Intronn, Inc. Spliceosome mediated rna trans-splicing and correction of factor viii genetic defects using spliceosome mediated rna trans spling
EP1549755A4 (en) * 2002-06-05 2006-09-20 Intronn Inc SPREAD OSOM-MEDIATED TRANS-RNA SPLEISSEN IN STEM CELLS
US20040018622A1 (en) * 2002-07-17 2004-01-29 Mitchell Lloyd G. Spliceosome mediated RNA trans-splicing for correction of skin disorders
AU2003285009B2 (en) * 2002-10-23 2010-10-07 Virxsys Corporation Screening methods for identification of efficient pre-trans-splicing molecules
WO2004087958A1 (en) * 2003-03-31 2004-10-14 Council Of Scientific And Industrial Research Stn gene oligonucleotide primers for detecting salmonella species and detection prosses using the same
US8053232B2 (en) * 2004-01-23 2011-11-08 Virxsys Corporation Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing
US7968334B2 (en) * 2004-01-23 2011-06-28 Virxsys Corporation Expression of apoAI and variants thereof using spliceosome mediated RNA trans-splicing
CA2553828A1 (en) * 2004-01-23 2005-08-04 Intronn, Inc. 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
US7871795B2 (en) * 2004-10-08 2011-01-18 Virxsys Corporation Targeted trans-splicing of highly abundant transcripts for in vivo production of recombinant proteins
JP5017118B2 (ja) * 2004-10-08 2012-09-05 バークシス コーポレーション 組換えタンパク質をインビボ生産するための、非常に豊富な転写産物の標的化トランススプライシング
US7879321B2 (en) * 2004-10-08 2011-02-01 Virxsys Corporation Use of RNA trans-splicing for antibody gene transfer and antibody polypeptide production
DE102006009000B3 (de) * 2006-02-23 2007-09-06 Tutech Innovation Gmbh Rechengen
JP5081462B2 (ja) * 2007-02-02 2012-11-28 富士フイルム株式会社 トランススプライシング法による融合タンパク質作製方法
EP2151248A1 (en) * 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
EP2326711A2 (en) * 2008-08-20 2011-06-01 VIRxSYS Corporation Compositions and methods for generation of pluripotent stem cells
US8923125B2 (en) * 2008-10-24 2014-12-30 Qualcomm Incorporated Wireless network resource adaptation
ES2578993T3 (es) 2009-10-08 2016-08-03 Association Institut De Myologie Moléculas de ácido nucleico y métodos para intercambiar uno o más exones mediante corte y empalme en trans
WO2013016595A1 (en) 2011-07-28 2013-01-31 The Regents Of The University Of California Exonic splicing enhancers and exonic splicing silencers
WO2014045283A1 (en) * 2012-09-24 2014-03-27 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Restoration of the cftr function by splicing modulation
US9447411B2 (en) 2013-01-14 2016-09-20 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)
KR20250150157A (ko) 2018-04-17 2025-10-17 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 트랜스-스플라이싱 분자
EP4013873A1 (en) 2019-08-16 2022-06-22 Massachusetts Institute Of Technology Targeted trans-splicing using crispr/cas13
EP4522758A2 (en) 2022-05-13 2025-03-19 Ascidian Therapeutics, Inc. Abca4 trans-splicing molecules
EP4590825A1 (en) * 2022-09-22 2025-07-30 Rna Morph Ltd Folding oligonucleotides
EP4594505A1 (en) 2022-09-30 2025-08-06 Centre National de la Recherche Scientifique Therapy by trans-splicing of opa1 pre-messenger rnas for the treatment of diseases associated with opa1 gene mutations
CN120641122A (zh) 2022-11-23 2025-09-12 安铂生物公司 基因修饰核酸内切酶
WO2025128343A1 (en) 2023-12-11 2025-06-19 Just-Evotec Biologics, Inc. Protein expression using trans-splicing and split selectable markers

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ241310A (en) * 1991-01-17 1995-03-28 Gen Hospital Corp Trans-splicing ribozymes
NZ241311A (en) * 1991-01-17 1995-03-28 Gen Hospital Corp Rna sequence having trans-splicing activity, plant strains
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

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Publication number Publication date
EP1105513A2 (en) 2001-06-13
EP1489185A1 (en) 2004-12-22
WO2000009734A2 (en) 2000-02-24
JP4353390B2 (ja) 2009-10-28
WO2000009734A3 (en) 2000-05-18
CA2339300A1 (en) 2000-02-24
IL141409A0 (en) 2002-03-10
AU773186B2 (en) 2004-05-20
JP2007312780A (ja) 2007-12-06
AU5673599A (en) 2000-03-06
JP2004504001A (ja) 2004-02-12
US6280978B1 (en) 2001-08-28
WO2000009734A9 (en) 2000-07-13

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