AU2019406702B2 - An optimized acceptor splice site module for biological and biotechnological applications - Google Patents

An optimized acceptor splice site module for biological and biotechnological applications

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Publication number
AU2019406702B2
AU2019406702B2 AU2019406702A AU2019406702A AU2019406702B2 AU 2019406702 B2 AU2019406702 B2 AU 2019406702B2 AU 2019406702 A AU2019406702 A AU 2019406702A AU 2019406702 A AU2019406702 A AU 2019406702A AU 2019406702 B2 AU2019406702 B2 AU 2019406702B2
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Australia
Prior art keywords
sequence
nucleotides
nucleic acid
acceptor splice
interest
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AU2019406702A
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AU2019406702A1 (en
Inventor
Elvir BECIROVIC
Martin Biel
Stylianos MICHALAKIS
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Vigeneron GmbH
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Vigeneron GmbH
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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)
AU2019406702A 2018-12-20 2019-12-19 An optimized acceptor splice site module for biological and biotechnological applications Active AU2019406702B2 (en)

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 (2)

Publication Number Publication Date
AU2019406702A1 AU2019406702A1 (en) 2021-06-10
AU2019406702B2 true AU2019406702B2 (en) 2025-12-18

Family

ID=64755152

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2019406702A Active AU2019406702B2 (en) 2018-12-20 2019-12-19 An optimized acceptor splice site module for biological and biotechnological applications

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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020193580A1 (en) * 1995-12-15 2002-12-19 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
WO2014170480A1 (en) * 2013-04-18 2014-10-23 Fondazione Telethon Effective delivery of large genes by dual aav vectors

Family Cites Families (14)

* 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
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
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
GB9906615D0 (en) 1999-03-22 1999-05-19 Oxford Biomedica Ltd Vector
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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020193580A1 (en) * 1995-12-15 2002-12-19 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
WO2014170480A1 (en) * 2013-04-18 2014-10-23 Fondazione Telethon Effective delivery of large genes by dual aav vectors

Also Published As

Publication number Publication date
IL284021A (en) 2021-08-31
CN113474460A (zh) 2021-10-01
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
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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