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 (enExample
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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
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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
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
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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
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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
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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
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    • 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)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (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
CN2021075874 2021-02-07
CNPCT/CN2021/075874 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 (enExample) 2025-02-18

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

Families Citing this family (6)

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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
WO2024120528A1 (en) * 2022-12-08 2024-06-13 Huidagene Therapeutics (Singapore) Pte. Ltd. Improved system for producing rna-packaged aav particles
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
WO2026095803A1 (en) * 2024-11-04 2026-05-07 Umc Utrecht Holding B.V. Sirna delivery with biological vectors

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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JP6738728B2 (ja) * 2013-06-17 2020-08-19 ザ・ブロード・インスティテュート・インコーポレイテッド 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
US11352647B2 (en) * 2016-08-17 2022-06-07 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
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
CN111328290A (zh) * 2017-06-26 2020-06-23 博德研究所 用于靶向核酸编辑的基于crispr/cas-腺嘌呤脱氨酶的组合物、系统和方法
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
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Patent 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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IL305002A (en) 2023-10-01
US12031148B2 (en) 2024-07-09
WO2022166954A1 (en) 2022-08-11
US20240368629A1 (en) 2024-11-07
EP4288112A1 (en) 2023-12-13
EP4288112A4 (en) 2025-08-06
KR20230142776A (ko) 2023-10-11
BR112023015769A2 (pt) 2023-12-12
CN115209924A (zh) 2022-10-18
US20230062529A1 (en) 2023-03-02
CA3207601A1 (en) 2022-08-11
AU2022217349A1 (en) 2023-08-31

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