JP7504458B2 - Ａａｖ形質導入と関連付けられる自然免疫応答の阻害のための方法および組成物 - Google Patents

Ａａｖ形質導入と関連付けられる自然免疫応答の阻害のための方法および組成物 Download PDF

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JP7504458B2
JP7504458B2 JP2020539080A JP2020539080A JP7504458B2 JP 7504458 B2 JP7504458 B2 JP 7504458B2 JP 2020539080 A JP2020539080 A JP 2020539080A JP 2020539080 A JP2020539080 A JP 2020539080A JP 7504458 B2 JP7504458 B2 JP 7504458B2
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リー，チェンウェン
サムルスキ，リチャード・ジュード
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ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル
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    • 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
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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  • Animal Behavior & Ethology (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2020539080A 2018-01-19 2019-01-18 Ａａｖ形質導入と関連付けられる自然免疫応答の阻害のための方法および組成物 Active JP7504458B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862619468P 2018-01-19 2018-01-19
US62/619,468 2018-01-19
PCT/US2019/014211 WO2019143950A2 (en) 2018-01-19 2019-01-18 Methods and compositions for inhibition of innate immune response associated with aav transduction

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JP2021511035A JP2021511035A (ja) 2021-05-06
JP2021511035A5 JP2021511035A5 (https=) 2022-02-24
JPWO2019143950A5 JPWO2019143950A5 (https=) 2022-02-24
JP7504458B2 true JP7504458B2 (ja) 2024-06-24

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US (1) US20200340013A1 (https=)
EP (1) EP3740572A4 (https=)
JP (1) JP7504458B2 (https=)
CN (1) CN111918966B (https=)
AU (1) AU2019210190B2 (https=)
CA (1) CA3088538A1 (https=)
WO (1) WO2019143950A2 (https=)

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AU2020270960B2 (en) 2019-04-12 2026-01-29 Ultragenyx Pharmaceutical Inc. Engineered producer cell lines and methods of making and using the same
ES2827599B2 (es) * 2019-11-21 2022-05-05 Fundacion Para La Investigacion Biomedica Del Hospital Univ Nino Jesus Celulas madre mesenquimales para ser usadas como vehiculos de agentes terapeuticos
US20220106609A1 (en) * 2019-12-17 2022-04-07 Nikegen, Llc Paravoviral vectors and methods of making and use thereof
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
US20250019668A1 (en) * 2021-10-29 2025-01-16 Ultragenyx Pharmaceutical Inc. Engineered cell lines for increased production of recombinant adeno-associated virus (raav)
WO2024123813A1 (en) * 2022-12-05 2024-06-13 The Trustees Of Columbia University In The City Of New York Viral vectors for increasing the specificity of gene expression
CN117701638B (zh) * 2023-12-18 2024-10-18 苏州新芽基因生物技术有限公司 一种抑制残留aav载体质粒和质粒骨架转录活性的方法

Citations (2)

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US20110070241A1 (en) 2009-06-30 2011-03-24 Duke University Methods for modulating immune responses to aav gene therapy vectors
WO2016110518A1 (en) 2015-01-07 2016-07-14 Universitat Autònoma De Barcelona Single-vector gene construct comprising insulin and glucokinase genes

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DE19959045A1 (de) * 1999-12-07 2001-06-28 Deutsches Krebsforsch MCP-1 exprimierende AAV-Expressionsvektoren und deren therapeutische Verwendung
US20110206639A1 (en) * 2004-04-15 2011-08-25 Christie Vermeulen Replication competent viruses capable of silencing virus inhibitory factor expression
WO2014145968A2 (en) * 2013-03-15 2014-09-18 The Regents Of The University Of California Rna interference functions as an antiviral immunity in mammals
US20190000943A1 (en) * 2015-08-13 2019-01-03 Aavet Therapeutics, Llc Aav6 vectors for immunotherapy

Patent Citations (2)

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Publication number Priority date Publication date Assignee Title
US20110070241A1 (en) 2009-06-30 2011-03-24 Duke University Methods for modulating immune responses to aav gene therapy vectors
WO2016110518A1 (en) 2015-01-07 2016-07-14 Universitat Autònoma De Barcelona Single-vector gene construct comprising insulin and glucokinase genes

Non-Patent Citations (4)

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Title
Shao W. et al.，Double-stranded RNA innate immune response activation from long-term adeno-associated virus vector transduction，JCI Insight，2018年07月21日，Vol. 3(12):e120474
Soulat D. et al.，Cytoplasmic Listeria monocytogenes stimulates IFN-beta synthesis without requiring the adapter protein MAVS，FEBS Lett，2006年，Vol. 580，pp. 2341-2346
Sugiyama T,. et al.，Mechanism of inhibition of lipopolysaccharide-induced interferon-β production by 2-aminopurine，Mol Immunol，2012年，Vol. 52，pp. 299-304
佐藤 文彦 ほか，一過的ＲＮＡｉ系の開発，化学と生物，2005年，Vol. 43，pp. 177-183

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Publication number Publication date
CA3088538A1 (en) 2019-07-25
JP2021511035A (ja) 2021-05-06
EP3740572A2 (en) 2020-11-25
EP3740572A4 (en) 2022-01-05
CN111918966B (zh) 2024-12-17
US20200340013A1 (en) 2020-10-29
AU2019210190B2 (en) 2025-06-05
AU2019210190A1 (en) 2020-08-06
CN111918966A (zh) 2020-11-10
WO2019143950A3 (en) 2019-09-06
WO2019143950A2 (en) 2019-07-25

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