CN113474460A - 用于生物学应用和生物技术应用的优化的受体剪接位点模块 - Google Patents

用于生物学应用和生物技术应用的优化的受体剪接位点模块 Download PDF

Info

Publication number
CN113474460A
CN113474460A CN201980092298.4A CN201980092298A CN113474460A CN 113474460 A CN113474460 A CN 113474460A CN 201980092298 A CN201980092298 A CN 201980092298A CN 113474460 A CN113474460 A CN 113474460A
Authority
CN
China
Prior art keywords
sequence
nucleic acid
nucleotides
region
splice site
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CN201980092298.4A
Other languages
English (en)
Chinese (zh)
Inventor
斯蒂利亚诺斯·米查拉基斯
马丁·比尔
埃尔维尔·贝西洛维奇
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Vignero Co ltd
Original Assignee
Vignero Co ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Vignero Co ltd filed Critical Vignero Co ltd
Publication of CN113474460A publication Critical patent/CN113474460A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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

Landscapes

  • 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)
CN201980092298.4A 2018-12-20 2019-12-19 用于生物学应用和生物技术应用的优化的受体剪接位点模块 Pending CN113474460A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Publications (1)

Publication Number Publication Date
CN113474460A true CN113474460A (zh) 2021-10-01

Family

ID=64755152

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201980092298.4A Pending CN113474460A (zh) 2018-12-20 2019-12-19 用于生物学应用和生物技术应用的优化的受体剪接位点模块

Country Status (10)

Country Link
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=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11202106356QA (en) * 2018-12-20 2021-07-29 Vigeneron Gmbh An optimized acceptor splice site module for biological and biotechnological applications
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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000056910A1 (en) * 1999-03-22 2000-09-28 Oxford Biomedica (Uk) Limited Retroviral vectors comprising functional and non-functional splice donor and splice acceptor sites
US6436707B1 (en) * 1998-03-27 2002-08-20 Lexicon Genetics Incorporated Vectors for gene mutagenesis and gene discovery
US20030077754A1 (en) * 1995-12-15 2003-04-24 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
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
CN105408352A (zh) * 2013-04-18 2016-03-16 泰莱托恩基金会 通过双重aav载体有效递送大基因

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US20020193580A1 (en) * 1995-12-15 2002-12-19 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
CA2477295A1 (en) 2002-02-25 2003-09-04 Intronn, Inc. Trans-splicing mediated imaging of gene expression
US7968334B2 (en) 2004-01-23 2011-06-28 Virxsys Corporation Expression of apoAI and variants thereof using spliceosome mediated RNA trans-splicing
US9303078B2 (en) 2009-10-08 2016-04-05 Association Institut De Myologie Nucleic acid molecules and methods for exchanging exon(s) by transsplicing
SG2014012207A (en) 2011-08-12 2014-05-29 Virxsys Corp Compositions and methods for inducing apoptosis
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
JP7007273B2 (ja) * 2015-12-22 2022-01-24 アンセルム（アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル） 遺伝子治療用の改良された複合型二重組換えａａｖベクターシステム
US12043848B2 (en) 2017-10-16 2024-07-23 Vigeneron Gmbh AAV vectors
SG11202106356QA (en) * 2018-12-20 2021-07-29 Vigeneron Gmbh An optimized acceptor splice site module for biological and biotechnological applications

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030077754A1 (en) * 1995-12-15 2003-04-24 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US6436707B1 (en) * 1998-03-27 2002-08-20 Lexicon Genetics Incorporated Vectors for gene mutagenesis and gene discovery
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
WO2000056910A1 (en) * 1999-03-22 2000-09-28 Oxford Biomedica (Uk) Limited Retroviral vectors comprising functional and non-functional splice donor and splice acceptor sites
CN105408352A (zh) * 2013-04-18 2016-03-16 泰莱托恩基金会 通过双重aav载体有效递送大基因
US20160076054A1 (en) * 2013-04-18 2016-03-17 Fondazione Telethon Effective delivery of large genes by dual aav vectors

Also Published As

Publication number Publication date
IL284021A (en) 2021-08-31
US20220160898A1 (en) 2022-05-26
JP2022515606A (ja) 2022-02-21
JP7668737B2 (ja) 2025-04-25
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

Similar Documents

Publication Publication Date Title
KR102938671B1 (ko) 생물학적 및 생물공학적 적용에 최적화된 수여 스플라이싱 부위 모듈
US20240318149A1 (en) AAV Vectors
AU2017322377B2 (en) Acid-alpha glucosidase variants and uses thereof
US11459558B2 (en) Methods for using transcription-dependent directed evolution of AAV capsids
CN115427578B (zh) 基因疗法
AU2019255708B2 (en) Trans-splicing molecules
CN114126665B (zh) 眼底黄色斑点症(abca4)的基因疗法
CN113710693B (zh) Dna结合结构域反式激活因子及其用途
EP3737423B1 (en) Compositions and methods for treating retinal disorders
WO2024069144A1 (en) Rna editing vector
CA3218631A1 (en) Vector system
RU2811724C2 (ru) РЕДАКТИРОВАНИЕ ГЕНОВ С ИСПОЛЬЗОВАНИЕМ МОДИФИЦИРОВАННОЙ ДНК С ЗАМКНУТЫМИ КОНЦАМИ (зкДНК)
RU2793112C2 (ru) Векторы aav
CN117836420A (zh) 重组tert编码病毒基因组和运载体
OA21893A (en) KCNV2 variants and their use.
HK40030486A (en) Compositions and methods for treating retinal disorders
HK40030486B (en) Compositions and methods for treating retinal disorders

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination