JP6929230B2 - スペーサーを含む核酸分子およびその使用の方法 - Google Patents

スペーサーを含む核酸分子およびその使用の方法 Download PDF

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JP6929230B2
JP6929230B2 JP2017559315A JP2017559315A JP6929230B2 JP 6929230 B2 JP6929230 B2 JP 6929230B2 JP 2017559315 A JP2017559315 A JP 2017559315A JP 2017559315 A JP2017559315 A JP 2017559315A JP 6929230 B2 JP6929230 B2 JP 6929230B2
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JP2018515102A (ja
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グレイ，ジョン，ティー．
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セント ジュード チルドレンズ リサーチ ホスピタル インコーポレイテッド
セント ジュード チルドレンズ リサーチ ホスピタル インコーポレイテッド
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    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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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
    • C12N15/86—Viral vectors
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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
    • C12N15/86—Viral vectors
    • C12N15/861—Adenoviral vectors
    • C12N15/8616—Special methods for targeting systems
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/17—Immunomodulatory nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer

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  • Health & Medical Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • Microbiology (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
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  • Veterinary Medicine (AREA)
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  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Manufacturing & Machinery (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2017559315A 2015-05-14 2016-05-13 スペーサーを含む核酸分子およびその使用の方法 Active JP6929230B2 (ja)

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JP2021130440A JP7316326B2 (ja) 2015-05-14 2021-08-10 スペーサーを含む核酸分子およびその使用の方法
JP2023115773A JP2023138527A (ja) 2015-05-14 2023-07-14 スペーサーを含む核酸分子およびその使用の方法

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US201562161505P 2015-05-14 2015-05-14
US62/161,505 2015-05-14
PCT/US2016/032321 WO2016183422A1 (en) 2015-05-14 2016-05-13 Nucleic acid molecules containing spacers and methods of use thereof

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JP2021130440A Active JP7316326B2 (ja) 2015-05-14 2021-08-10 スペーサーを含む核酸分子およびその使用の方法
JP2023115773A Pending JP2023138527A (ja) 2015-05-14 2023-07-14 スペーサーを含む核酸分子およびその使用の方法

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US (2) US11103597B2 (enExample)
EP (1) EP3294309A4 (enExample)
JP (3) JP6929230B2 (enExample)
AU (3) AU2016260401B2 (enExample)
WO (1) WO2016183422A1 (enExample)

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AU2016260401B2 (en) 2015-05-14 2022-05-19 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers and methods of use thereof
EP3361869B1 (en) * 2015-10-14 2025-12-03 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
US20200113972A1 (en) * 2017-04-14 2020-04-16 Rhode Island Hospital Vegf gene therapy for tendon and ligament injuries
MY203184A (en) 2017-08-09 2024-06-13 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
US20210002667A1 (en) * 2018-03-15 2021-01-07 Intergalactic Therapeutics, Inc. Synthetic dna vectors and methods of use
MX2021001599A (es) 2018-08-09 2021-07-02 Bioverativ Therapeutics Inc Moleculas de acido nucleico y sus usos para la terapia genica no viral.
AU2019363593A1 (en) 2018-10-25 2021-04-29 Takeda Pharmaceutical Company Limited AAV triple-plasmid system
CN115066255A (zh) 2019-09-18 2022-09-16 星际治疗有限公司 合成的dna载体和使用方法
CN115197967B (zh) * 2021-04-08 2023-09-15 广州派真生物技术有限公司 制备重组腺相关病毒的辅助质粒及其应用

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US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
CA2264499A1 (en) * 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
WO2001036623A2 (en) 1999-11-05 2001-05-25 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
US20020182595A1 (en) 2001-04-27 2002-12-05 Weitzman Matthew D. Method of identifying cellular regulators of adeno-associated virus (AAV)
WO2002097056A2 (en) 2001-05-31 2002-12-05 The Rockefeller University Method for generating replication defective viral vectors that are helper free
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
CA2500397A1 (en) 2002-09-26 2004-04-08 Greenville Hospital System Aav itr-mediated modulation
ES2381796T3 (es) * 2004-09-22 2012-05-31 St. Jude Children's Research Hospital Expresión mejorada de Factor IX en vectores de terapia génica
EP2524037B1 (en) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
US9249425B2 (en) 2011-05-16 2016-02-02 The Trustees Of The University Of Pennslyvania Proviral plasmids and production of recombinant adeno-associated virus
TWI702955B (zh) * 2012-05-15 2020-09-01 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒（ａａｖ）ｓｆｌｔ-１治療老年性黃斑部退化（ａｍｄ）
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BR112015021884A8 (pt) 2013-03-15 2019-11-26 Childrens Hospital Philadelphia vetores de plasmídeo recombinantes, partícula viral avv ou pluralidade de partículas virais, seus métodos de produção e seus usos, e composição farmacêutica
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AU2016260401B2 (en) 2015-05-14 2022-05-19 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers and methods of use thereof

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Publication number Publication date
US20180126006A1 (en) 2018-05-10
AU2025203194A1 (en) 2025-05-22
JP2021184734A (ja) 2021-12-09
US11103597B2 (en) 2021-08-31
EP3294309A1 (en) 2018-03-21
JP2023138527A (ja) 2023-10-02
JP7316326B2 (ja) 2023-07-27
AU2022215274A1 (en) 2022-09-01
EP3294309A4 (en) 2019-01-16
WO2016183422A1 (en) 2016-11-17
AU2016260401B2 (en) 2022-05-19
CA2983593A1 (en) 2016-11-17
US20210369869A1 (en) 2021-12-02
US12453783B2 (en) 2025-10-28
JP2018515102A (ja) 2018-06-14
AU2016260401A1 (en) 2017-11-02

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