CN107532173A - 靶向中枢神经系统的aav载体 - Google Patents

靶向中枢神经系统的aav载体 Download PDF

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Publication number
CN107532173A
CN107532173A CN201580074023.XA CN201580074023A CN107532173A CN 107532173 A CN107532173 A CN 107532173A CN 201580074023 A CN201580074023 A CN 201580074023A CN 107532173 A CN107532173 A CN 107532173A
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Prior art keywords
aav
nucleic acid
particle
capsid
aav capsid
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Pending
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CN201580074023.XA
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Chinese (zh)
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S.格雷
T.麦考恩
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University of North Carolina at Chapel Hill
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University of North Carolina at Chapel Hill
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Priority to CN202210236316.5A priority Critical patent/CN114480440A/zh
Publication of CN107532173A publication Critical patent/CN107532173A/zh
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    • A—HUMAN NECESSITIES
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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CN201580074023.XA 2014-11-21 2015-11-20 靶向中枢神经系统的aav载体 Pending CN107532173A (zh)

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Application Number Priority Date Filing Date Title
CN202210236316.5A CN114480440A (zh) 2014-11-21 2015-11-20 靶向中枢神经系统的aav载体

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US201462082897P 2014-11-21 2014-11-21
US62/082897 2014-11-21
US201562218857P 2015-09-15 2015-09-15
US62/218857 2015-09-15
PCT/US2015/061788 WO2016081811A1 (en) 2014-11-21 2015-11-20 Aav vectors targeted to the central nervous system

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US (4) US10532110B2 (enExample)
EP (2) EP4344741A3 (enExample)
JP (4) JP2017536116A (enExample)
KR (2) KR20240063169A (enExample)
CN (2) CN114480440A (enExample)
AU (2) AU2015349759B2 (enExample)
BR (1) BR112017010447A2 (enExample)
CA (1) CA2967393A1 (enExample)
IL (1) IL252072B (enExample)
MX (1) MX2017006652A (enExample)
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WO2019223740A1 (zh) * 2018-05-24 2019-11-28 北京肽和生物科技有限公司 一种靶向肿瘤细胞的嵌合肽或嵌合蛋白及其应用
CN111574595A (zh) * 2020-05-22 2020-08-25 中国医学科学院血液病医院(中国医学科学院血液学研究所) 腺相关病毒突变体及其应用
CN112272672A (zh) * 2018-04-03 2021-01-26 斯特里迪比奥公司 抗体逃避性病毒载体
CN112566923A (zh) * 2018-06-12 2021-03-26 北卡罗来纳大学教堂山分校 合成嗜肝性腺相关病毒衣壳及其用途
CN113166208A (zh) * 2018-10-02 2021-07-23 沃雅戈治疗公司 重新定向aav衣壳的嗜性
CN113423434A (zh) * 2018-12-05 2021-09-21 阿贝奥纳治疗有限公司 用于基因递送的重组腺相关病毒载体
CN113563430A (zh) * 2021-07-30 2021-10-29 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
CN115210248A (zh) * 2020-02-18 2022-10-18 北卡罗来纳大学查佩尔希尔分校 Aav衣壳-启动子相互作用和细胞选择性基因表达
CN115916986A (zh) * 2020-05-05 2023-04-04 北卡罗来纳大学查佩尔希尔分校 修饰的腺相关病毒5衣壳及其用途

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IL313246B2 (en) 2018-06-28 2025-10-01 Univ North Carolina Chapel Hill Optimal cln5 expression genes and cassettes and their use
IL280300B2 (en) 2018-08-10 2025-06-01 Univ North Carolina Chapel Hill Optimized cln7 genes and expression cassettes and their use
CN113966399A (zh) 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
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