CN1209068A - 作为基因治疗所用的dna载体的透明质酸及治疗异常视网膜血管化的vegf反义dna - Google Patents

作为基因治疗所用的dna载体的透明质酸及治疗异常视网膜血管化的vegf反义dna Download PDF

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CN1209068A
CN1209068A CN96199290A CN96199290A CN1209068A CN 1209068 A CN1209068 A CN 1209068A CN 96199290 A CN96199290 A CN 96199290A CN 96199290 A CN96199290 A CN 96199290A CN 1209068 A CN1209068 A CN 1209068A
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composition
sequence
virus
vegf
antisense
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Pending
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CN96199290A
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English (en)
Chinese (zh)
Inventor
波罗斯卡·伊丽莎白·雷考茨
伊恩·杰弗里·康斯特伯
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Alchemia Oncology Pty Ltd
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Hyal Pharmaceutical Australia Ltd
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Priority claimed from AUPN6161A external-priority patent/AUPN616195A0/en
Priority claimed from AUPN9047A external-priority patent/AUPN904796A0/en
Application filed by Hyal Pharmaceutical Australia Ltd filed Critical Hyal Pharmaceutical Australia Ltd
Publication of CN1209068A publication Critical patent/CN1209068A/zh
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    • 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
    • C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • 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
    • C12N15/1137—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 against enzymes
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/022—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from an adenovirus
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    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

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  • Inorganic Chemistry (AREA)
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  • Dermatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Ophthalmology & Optometry (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CN96199290A 1995-10-23 1996-10-22 作为基因治疗所用的dna载体的透明质酸及治疗异常视网膜血管化的vegf反义dna Pending CN1209068A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
AUPN6161 1995-10-23
AUPN6161A AUPN616195A0 (en) 1995-10-23 1995-10-23 Method and composition for treatment of ocular diseases
AUPN9047 1996-04-01
AUPN9047A AUPN904796A0 (en) 1996-04-01 1996-04-01 Method and composition for treatment of ocular diseases

Publications (1)

Publication Number Publication Date
CN1209068A true CN1209068A (zh) 1999-02-24

Family

ID=25645043

Family Applications (1)

Application Number Title Priority Date Filing Date
CN96199290A Pending CN1209068A (zh) 1995-10-23 1996-10-22 作为基因治疗所用的dna载体的透明质酸及治疗异常视网膜血管化的vegf反义dna

Country Status (8)

Country Link
EP (1) EP0859636A4 (de)
JP (1) JP2000507915A (de)
KR (1) KR19990066967A (de)
CN (1) CN1209068A (de)
CA (1) CA2235685A1 (de)
MY (1) MY134778A (de)
NZ (1) NZ320006A (de)
WO (1) WO1997015330A1 (de)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007006232A1 (fr) * 2005-07-13 2007-01-18 Peixue Ling Complexe de phospholipide et d’acide hyaluronique et procédé de préparation

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6475795B1 (en) 1991-07-03 2002-11-05 Meditech Research, Ltd. Use of hyaluronan in gene therapy
US6875753B1 (en) 1996-03-14 2005-04-05 The Governors Of The University Of Alberta Methods for cell mobilization using in vivo treatment with hyaluronan (HA)
EP0963196B1 (de) * 1996-09-27 2005-01-19 Jagotec Ag Arzneistoffverabreichungssystem mit hyaluronsäure
GB9723780D0 (en) * 1997-11-12 1998-01-07 Univ Manchester Regulation of ocular angiogenesis
SE9904121D0 (sv) * 1999-11-15 1999-11-15 Gustaf Jederstroem Hydrophobe biomolecular structure
IL150485A0 (en) * 1999-12-28 2002-12-01 Novartis Ag Method of achieving persistent transgene expression
AU2001264317A1 (en) * 2000-06-20 2002-01-02 Koken Co., Ltd. Preparations for oligonucleotide transfer
EP1407787B1 (de) 2001-06-20 2009-04-29 Dainippon Sumitomo Pharma Co., Ltd. Verfahren zur förderung des nukleinsäuretransfers
EP1480656A4 (de) * 2002-02-15 2007-07-18 Res Dev Foundation Hyaluronsäure-vermittelte adenovirus-transduktion
WO2004105784A1 (en) * 2003-05-29 2004-12-09 The University Of Manchester Class iii slrp agonists for the reduction of blood vessel formation
CA2582997C (en) * 2004-10-05 2015-01-13 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Method and apparatus for screening for retinopathy
MX2007011735A (es) 2005-03-24 2008-03-14 Thrombogenics Nv Anticuerpo anti-plgf novedoso.
EP1904095B1 (de) 2005-06-30 2013-05-29 VIB, vzw Behandlung von leberzirrhose und ihre komplikationen
CN102239178B (zh) 2008-10-02 2017-06-23 Vib研究所 抑制PlGF以治疗费城染色体阳性白血病
AU2012321082B2 (en) 2011-12-01 2015-03-12 Oxurion NV Improving trabeculectomy outcome
KR101420751B1 (ko) * 2011-12-12 2014-07-17 서강대학교산학협력단 망막 질환 치료제 후보물질의 스크리닝 방법
WO2021243264A2 (en) * 2020-05-28 2021-12-02 Lonza Ltd Formulations for viral vectors

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2636339B1 (fr) * 1988-09-09 1992-07-17 Auge Pier Gel aqueux a base d'acide hyaluronique et d'acide desoxyribonucleique utilisable en cosmetique, et procede de preparation
CA1340994C (en) * 1989-09-21 2000-05-16 Rudolf Edgar Dr. Falk Treatment of conditions and disease
AU1469292A (en) * 1991-01-18 1992-08-27 Oncogene Science, Inc. Methods of transcriptionally modulating expression of growth factor genes and growth factor receptor genes
US5731294A (en) * 1993-07-27 1998-03-24 Hybridon, Inc. Inhibition of neovasularization using VEGF-specific oligonucleotides
US5641756A (en) * 1993-07-27 1997-06-24 Hybridon, Inc. Modified VEGF oligonucleotides
US6410322B1 (en) * 1993-07-27 2002-06-25 Hybridon Inc Antisense oligonucleotide inhibition of vascular endothelial growth factor expression
US6150092A (en) * 1994-06-27 2000-11-21 Taogosei Company, Ltd. Antisense nucleic acid compound targeted to VEGF

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007006232A1 (fr) * 2005-07-13 2007-01-18 Peixue Ling Complexe de phospholipide et d’acide hyaluronique et procédé de préparation

Also Published As

Publication number Publication date
EP0859636A1 (de) 1998-08-26
CA2235685A1 (en) 1997-05-01
MY134778A (en) 2007-12-31
KR19990066967A (ko) 1999-08-16
JP2000507915A (ja) 2000-06-27
EP0859636A4 (de) 2002-05-02
WO1997015330A1 (en) 1997-05-01
NZ320006A (en) 2001-12-21

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