CN110691846A - 合理的多倍体腺相关病毒载体及其制造和使用方法 - Google Patents

合理的多倍体腺相关病毒载体及其制造和使用方法 Download PDF

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CN110691846A
CN110691846A CN201880032343.2A CN201880032343A CN110691846A CN 110691846 A CN110691846 A CN 110691846A CN 201880032343 A CN201880032343 A CN 201880032343A CN 110691846 A CN110691846 A CN 110691846A
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aav
serotype
virions
nucleic acid
capsid
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Chinese (zh)
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C.李
R.J.萨马尔斯基
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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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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A01K2267/03—Animal model, e.g. for test or diseases
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
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CN201880032343.2A 2018-05-07 2018-07-31 合理的多倍体腺相关病毒载体及其制造和使用方法 Pending CN110691846A (zh)

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US201862668056P 2018-05-07 2018-05-07
US62/668056 2018-05-07
US201862678675P 2018-05-31 2018-05-31
US62/678675 2018-05-31
PCT/US2018/044632 WO2019216932A1 (en) 2018-05-07 2018-07-31 Rational polyploid adeno-associated virus vectors and methods of making and using the same

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JP (2) JP2021522775A (https=)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110684800A (zh) * 2018-11-02 2020-01-14 深圳益世康宁生物科技有限公司 一种携带肿瘤-睾丸抗原10基因的重组腺相关病毒载体及其应用价值

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021142130A1 (en) * 2020-01-07 2021-07-15 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats and methods of their use as promoters
CN116096734A (zh) * 2020-05-13 2023-05-09 沃雅戈治疗公司 Aav衣壳的向性的重定向
JP2024515626A (ja) * 2021-04-16 2024-04-10 アスクレピオス バイオファーマシューティカル， インコーポレイテッド 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドａａｖビリオン
AU2022286647A1 (en) * 2021-06-02 2023-11-30 Uniqure Biopharma B.V. Insect cell production of parvoviral vectors with modified capsid proteins

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WO2004099423A1 (en) * 2003-05-01 2004-11-18 University Of Florida Research Foundation, Inc. Vp2-modified raav vectors and uses thereof
WO2012112578A2 (en) * 2011-02-14 2012-08-23 The Children's Hospital Of Philadelphia Improved aav8 vector with enhanced functional activity and methods of use thereof
US20150023924A1 (en) * 2013-07-22 2015-01-22 The Children's Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues

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EP1660123B1 (en) * 2003-09-02 2013-03-13 GlaxoSmithKline Biologicals s.a. Rotavirus vaccine
EP2848253A1 (en) * 2006-06-19 2015-03-18 Asklepios Biopharmaceutical, Inc. Modified factor VIII and factor IX genes and vectors for gene therapy
US10415056B2 (en) * 2010-11-10 2019-09-17 Fred Hutchinson Cancer Research Center Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination
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WO2018109207A1 (en) * 2016-12-16 2018-06-21 Uniqure Ip B.V. Immunoadsorption
CN110770346B (zh) * 2017-03-15 2024-01-12 北卡罗来纳-查佩尔山大学 多倍体腺相关病毒载体及其制备和使用方法

Patent Citations (3)

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Publication number Priority date Publication date Assignee Title
WO2004099423A1 (en) * 2003-05-01 2004-11-18 University Of Florida Research Foundation, Inc. Vp2-modified raav vectors and uses thereof
WO2012112578A2 (en) * 2011-02-14 2012-08-23 The Children's Hospital Of Philadelphia Improved aav8 vector with enhanced functional activity and methods of use thereof
US20150023924A1 (en) * 2013-07-22 2015-01-22 The Children's Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues

Non-Patent Citations (1)

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Title
S.P.BECERRA ET AL.: "Synthesis of Adeno-Associated Virus Structural Proteins Requires Both Alternative mRNA Splicing and Alternative Initiations from a Single Transcript", 《JOURNAL OF VIROLOGY》 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110684800A (zh) * 2018-11-02 2020-01-14 深圳益世康宁生物科技有限公司 一种携带肿瘤-睾丸抗原10基因的重组腺相关病毒载体及其应用价值

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IL269184A (en) 2019-11-28
AU2018422759B2 (en) 2026-01-22
EP3596203A1 (en) 2020-01-22
AU2018422759A1 (en) 2020-01-02
EP3596203A4 (en) 2021-12-01
IL269184B2 (en) 2025-08-01
IL269184B1 (en) 2025-04-01
JP2023154428A (ja) 2023-10-19
WO2019216932A1 (en) 2019-11-14
NZ759802A (en) 2025-08-29
JP2021522775A (ja) 2021-09-02
CN117535247A (zh) 2024-02-09

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