JP7668737B2 - 生物学的および生物工学的用途向けに最適化された受容スプライス部位モジュール - Google Patents

生物学的および生物工学的用途向けに最適化された受容スプライス部位モジュール Download PDF

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JP7668737B2
JP7668737B2 JP2021534372A JP2021534372A JP7668737B2 JP 7668737 B2 JP7668737 B2 JP 7668737B2 JP 2021534372 A JP2021534372 A JP 2021534372A JP 2021534372 A JP2021534372 A JP 2021534372A JP 7668737 B2 JP7668737 B2 JP 7668737B2
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sequence
nucleotides
nucleic acid
acceptor splice
splice site
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JP2022515606A5 (https=
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ミヒャラキス スタイリアノス
ビエル マルティン
ベツィロビク エルビール
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ビゲネロン ゲゼルシャフト ミット ベシュレンクテル ハフツング
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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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N15/63—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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021534372A 2018-12-20 2019-12-19 生物学的および生物工学的用途向けに最適化された受容スプライス部位モジュール Active JP7668737B2 (ja)

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JP2024229104A JP2025060848A (ja) 2018-12-20 2024-12-25 生物学的および生物工学的用途向けに最適化された受容スプライス部位モジュール

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EP18214415 2018-12-20
EP18214415.4 2018-12-20
PCT/EP2019/086454 WO2020127831A1 (en) 2018-12-20 2019-12-19 An optimized acceptor splice site module for biological and biotechnological applications

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JP7668737B2 true JP7668737B2 (ja) 2025-04-25

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JP2024229104A Pending JP2025060848A (ja) 2018-12-20 2024-12-25 生物学的および生物工学的用途向けに最適化された受容スプライス部位モジュール

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US (1) US12502438B2 (https=)
EP (1) EP3898996A1 (https=)
JP (2) JP7668737B2 (https=)
KR (1) KR102938671B1 (https=)
CN (1) CN113474460A (https=)
AU (1) AU2019406702B2 (https=)
CA (1) CA3122909A1 (https=)
IL (1) IL284021A (https=)
SG (1) SG11202106356QA (https=)
WO (1) WO2020127831A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
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JP2025060848A (ja) * 2018-12-20 2025-04-10 ビゲネロン ゲゼルシャフト ミット ベシュレンクテル ハフツング 生物学的および生物工学的用途向けに最適化された受容スプライス部位モジュール

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WO2020205604A1 (en) 2019-03-29 2020-10-08 Salk Institute For Biological Studies High-efficiency reconstitution of rna molecules
US20230287458A1 (en) * 2020-07-14 2023-09-14 Abeona Therapeutics Inc. Recombinant adeno-associated viral vectors for multipartite gene delivery
US20230374476A1 (en) * 2021-02-05 2023-11-23 University Of Massachusetts Prime editor system for in vivo genome editing
WO2022220968A1 (en) * 2021-04-15 2022-10-20 U1 Bio, Inc. High efficiency trans-splicing for replacement of targeted rna sequences in human cells
JP2024517939A (ja) * 2021-05-14 2024-04-23 ソーク インスティテュート フォー バイオロジカル スタディーズ 編集タンパク質の発現のための方法および組成物
CN116463380A (zh) * 2023-03-30 2023-07-21 中国科学院深圳先进技术研究院 一种重组腺相关病毒载体组合物、外源基因表达系统及方法

Citations (5)

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JP2002539797A (ja) 1999-03-22 2002-11-26 オックスフォード バイオメディカ（ユーケイ）リミテッド 機能的および非機能的なスプライス供与部位ならびにスプライス受容部位を含むレトロウイルスベクター
JP2002539764A (ja) 1998-11-20 2002-11-26 レキシコン ジェネティックス インコーポレーテッド 遺伝子トラッピングによる遺伝子突然変異誘発および遺伝子発見のためのベクター
JP2005518211A (ja) 2002-02-25 2005-06-23 イントロン，インコーポレーテッド 遺伝子発現のトランススプライシング媒介型イメージング
JP2009000120A (ja) 1998-08-13 2009-01-08 Virxsys Corp スプライソソーム媒介ｒｎａトランス−スプライシングにおける使用のための方法及び組成物
JP2016516424A (ja) 2013-04-18 2016-06-09 フォンダッツィオーネ・テレソン デュアルａａｖベクターによる大型遺伝子の効果的送達

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JP2009000120A (ja) 1998-08-13 2009-01-08 Virxsys Corp スプライソソーム媒介ｒｎａトランス−スプライシングにおける使用のための方法及び組成物
JP2002539764A (ja) 1998-11-20 2002-11-26 レキシコン ジェネティックス インコーポレーテッド 遺伝子トラッピングによる遺伝子突然変異誘発および遺伝子発見のためのベクター
JP2002539797A (ja) 1999-03-22 2002-11-26 オックスフォード バイオメディカ（ユーケイ）リミテッド 機能的および非機能的なスプライス供与部位ならびにスプライス受容部位を含むレトロウイルスベクター
JP2005518211A (ja) 2002-02-25 2005-06-23 イントロン，インコーポレーテッド 遺伝子発現のトランススプライシング媒介型イメージング
JP2016516424A (ja) 2013-04-18 2016-06-09 フォンダッツィオーネ・テレソン デュアルａａｖベクターによる大型遺伝子の効果的送達

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025060848A (ja) * 2018-12-20 2025-04-10 ビゲネロン ゲゼルシャフト ミット ベシュレンクテル ハフツング 生物学的および生物工学的用途向けに最適化された受容スプライス部位モジュール

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IL284021A (en) 2021-08-31
CN113474460A (zh) 2021-10-01
US20220160898A1 (en) 2022-05-26
JP2022515606A (ja) 2022-02-21
AU2019406702A1 (en) 2021-06-10
SG11202106356QA (en) 2021-07-29
KR20210125472A (ko) 2021-10-18
EP3898996A1 (en) 2021-10-27
AU2019406702B2 (en) 2025-12-18
KR102938671B1 (ko) 2026-03-11
CA3122909A1 (en) 2020-06-25
JP2025060848A (ja) 2025-04-10
WO2020127831A1 (en) 2020-06-25
US12502438B2 (en) 2025-12-23

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