JP2024508696A - Ｒｎａアデノ随伴ウイルス（ｒａａｖ）ベクター及びその用途 - Google Patents

Ｒｎａアデノ随伴ウイルス（ｒａａｖ）ベクター及びその用途 Download PDF

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JP2024508696A
JP2024508696A JP2023548288A JP2023548288A JP2024508696A JP 2024508696 A JP2024508696 A JP 2024508696A JP 2023548288 A JP2023548288 A JP 2023548288A JP 2023548288 A JP2023548288 A JP 2023548288A JP 2024508696 A JP2024508696 A JP 2024508696A
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sequence
rna
aav
virus
transcribed
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JP2024508696A5 (https=
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シャイ，リンユー
バイ，ウェイヤー
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フイダージーン・セラピューティクス（シンガポール）プライベート・リミテッド
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2750/14123—Virus like particles [VLP]
    • 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
    • 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
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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
    • 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • Physics & Mathematics (AREA)
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  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2023548288A 2021-02-07 2022-02-07 Ｒｎａアデノ随伴ウイルス（ｒａａｖ）ベクター及びその用途 Pending JP2024508696A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CNPCT/CN2021/075874 2021-02-07
CN2021075874 2021-02-07
PCT/CN2022/075366 WO2022166954A1 (en) 2021-02-07 2022-02-07 Rna adeno-associated virus (raav) vector and uses thereof

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JP2024508696A true JP2024508696A (ja) 2024-02-28
JP2024508696A5 JP2024508696A5 (https=) 2025-02-18

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US (2) US12031148B2 (https=)
EP (1) EP4288112A4 (https=)
JP (1) JP2024508696A (https=)
KR (1) KR20230142776A (https=)
CN (1) CN115209924A (https=)
AU (1) AU2022217349A1 (https=)
BR (1) BR112023015769A2 (https=)
CA (1) CA3207601A1 (https=)
IL (1) IL305002A (https=)
WO (1) WO2022166954A1 (https=)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116218904B (zh) * 2022-11-03 2025-04-15 国药中生生物技术研究院有限公司 一种可提高工程化细胞外泌体中特定核酸分子载量的方法及其应用
CN120659629A (zh) * 2022-12-08 2025-09-16 辉大基因治疗(新加坡)私人有限公司 用于产生rna包装的aav颗粒的改善的系统
CN117778431B (zh) * 2023-12-04 2024-10-25 广州派真生物技术有限公司 一种用于重组腺相关病毒包装的质粒系统及其应用
WO2025124549A1 (en) * 2023-12-13 2025-06-19 Huidagene Therapeutics (Singapore) Pte. Ltd. Improved system and method for producing recombinant dna-packaged aav particles
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 (4)

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US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
JP2018520686A (ja) * 2015-07-15 2018-08-02 ラトガース，ザ ステート ユニバーシティ オブ ニュージャージー ヌクレアーゼ非依存的な標的化遺伝子編集プラットフォームおよびその用途
JP2020503055A (ja) * 2017-01-05 2020-01-30 ラトガース，ザ ステート ユニバーシティ オブ ニュー ジャージー Ｄｎａ二本鎖切断に非依存的な標的化遺伝子編集プラットフォームおよびその用途
JP2020530992A (ja) * 2017-07-31 2020-11-05 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニーＳｉｇｍａ−Ａｌｄｒｉｃｈ Ｃｏ． ＬＬＣ Ｃｒｉｓｐｒ／ｃａｓアクチベーターシステムのための合成ガイドｒｎａ

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US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2018035387A1 (en) * 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
EP4485466A3 (en) * 2016-08-17 2025-04-02 The Broad Institute Inc. Novel crispr enzymes and systems
JP7215716B2 (ja) * 2017-01-13 2023-01-31 学校法人自治医科大学 肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのａａｖベクター
CN109055375B (zh) * 2018-07-26 2021-11-12 东南大学 一种crispr辅助反式增强子激活基因表达的方法及其应用
CN111349148A (zh) * 2020-03-13 2020-06-30 辉大(上海)生物科技有限公司 一种腺相关病毒载体及其用途
JP2023537296A (ja) * 2020-07-29 2023-08-31 ザ・キャソリック・ユニバーシティ・オブ・アメリカ ヒトゲノムリモデリングのためのバクテリオファージベースの人工ウイルスの設計

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
JP2018520686A (ja) * 2015-07-15 2018-08-02 ラトガース，ザ ステート ユニバーシティ オブ ニュージャージー ヌクレアーゼ非依存的な標的化遺伝子編集プラットフォームおよびその用途
JP2020503055A (ja) * 2017-01-05 2020-01-30 ラトガース，ザ ステート ユニバーシティ オブ ニュー ジャージー Ｄｎａ二本鎖切断に非依存的な標的化遺伝子編集プラットフォームおよびその用途
JP2020530992A (ja) * 2017-07-31 2020-11-05 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニーＳｉｇｍａ−Ａｌｄｒｉｃｈ Ｃｏ． ＬＬＣ Ｃｒｉｓｐｒ／ｃａｓアクチベーターシステムのための合成ガイドｒｎａ

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LIFE, vol. 10, 366, JPN6026000202, 2020, ISSN: 0005772360 *
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Publication number Publication date
KR20230142776A (ko) 2023-10-11
US12031148B2 (en) 2024-07-09
CA3207601A1 (en) 2022-08-11
EP4288112A4 (en) 2025-08-06
BR112023015769A2 (pt) 2023-12-12
CN115209924A (zh) 2022-10-18
US20230062529A1 (en) 2023-03-02
US20240368629A1 (en) 2024-11-07
IL305002A (en) 2023-10-01
WO2022166954A1 (en) 2022-08-11
AU2022217349A1 (en) 2023-08-31
EP4288112A1 (en) 2023-12-13

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