CN1329509A - 用于由靶细胞表达因子viii的腺伴随病毒载体 - Google Patents

用于由靶细胞表达因子viii的腺伴随病毒载体 Download PDF

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CN1329509A
CN1329509A CN99814128A CN99814128A CN1329509A CN 1329509 A CN1329509 A CN 1329509A CN 99814128 A CN99814128 A CN 99814128A CN 99814128 A CN99814128 A CN 99814128A CN 1329509 A CN1329509 A CN 1329509A
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factor viii
aav
factor
cell
sequence
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L·B·科特
P·C·克洛斯
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Avigen Inc
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Avigen Inc
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    • 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
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    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CN99814128A 1998-10-20 1999-10-19 用于由靶细胞表达因子viii的腺伴随病毒载体 Pending CN1329509A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US10499498P 1998-10-20 1998-10-20
US12597499P 1999-03-24 1999-03-24
US09/364,862 US6221349B1 (en) 1998-10-20 1999-07-30 Adeno-associated vectors for expression of factor VIII by target cells
US09/364,862 1999-07-30
US60/125,974 1999-07-30
US60/104,994 1999-07-30

Publications (1)

Publication Number Publication Date
CN1329509A true CN1329509A (zh) 2002-01-02

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CN99814128A Pending CN1329509A (zh) 1998-10-20 1999-10-19 用于由靶细胞表达因子viii的腺伴随病毒载体

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Country Link
US (1) US6221349B1 (https=)
EP (1) EP1124583A4 (https=)
JP (1) JP2002527493A (https=)
KR (1) KR20010082234A (https=)
CN (1) CN1329509A (https=)
AU (1) AU757688B2 (https=)
BR (1) BR9914643A (https=)
CA (1) CA2348068A1 (https=)
IL (1) IL142721A0 (https=)
MX (1) MXPA01004067A (https=)
WO (1) WO2000023116A1 (https=)

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CN103215308A (zh) * 2013-02-01 2013-07-24 中国科学院苏州生物医学院工程技术研究所 表达重组人fviii的整合质粒、细胞株及其构建方法和应用
CN104087613A (zh) * 2014-06-30 2014-10-08 中国科学院苏州生物医学工程技术研究所 基于aav-itr的基因表达微载体及其构建方法和应用
CN108093639A (zh) * 2015-04-16 2018-05-29 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
CN108368521A (zh) * 2015-08-06 2018-08-03 宾夕法尼亚州大学信托人 Glp-1和其在用于治疗代谢疾病的组合物中的用途
CN108602876A (zh) * 2015-11-13 2018-09-28 百深公司 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体
CN108884145A (zh) * 2015-11-13 2018-11-23 百深公司 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体
CN109328073A (zh) * 2016-02-23 2019-02-12 眼服务有限责任公司 用于治疗视网膜变性疾病的基因疗法
CN109562191A (zh) * 2016-04-15 2019-04-02 宾夕法尼亚州大学信托人 用于治疗血友病a的基因疗法
CN111876400A (zh) * 2020-08-06 2020-11-03 昆明理工大学 一种常温裂解酶Sly和编码此酶的多核苷酸
CN112384624A (zh) * 2018-05-15 2021-02-19 杜克大学 用于对阿克曼菌进行基因操纵的系统和方法
CN114269940A (zh) * 2019-07-03 2022-04-01 学校法人日本医科大学 用于产生包封核酸的aav中空颗粒的方法

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