SG11202106356QA - An optimized acceptor splice site module for biological and biotechnological applications - Google Patents

An optimized acceptor splice site module for biological and biotechnological applications

Info

Publication number
SG11202106356QA
SG11202106356QA SG11202106356QA SG11202106356QA SG11202106356QA SG 11202106356Q A SG11202106356Q A SG 11202106356QA SG 11202106356Q A SG11202106356Q A SG 11202106356QA SG 11202106356Q A SG11202106356Q A SG 11202106356QA SG 11202106356Q A SG11202106356Q A SG 11202106356QA
Authority
SG
Singapore
Prior art keywords
biological
splice site
acceptor splice
site module
biotechnological applications
Prior art date
Application number
SG11202106356QA
Other languages
English (en)
Inventor
Stylianos Michalakis
Martin Biel
Elvir Becirovic
Original Assignee
Vigeneron Gmbh
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 Vigeneron Gmbh filed Critical Vigeneron Gmbh
Publication of SG11202106356QA publication Critical patent/SG11202106356QA/en

Links

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)
SG11202106356QA 2018-12-20 2019-12-19 An optimized acceptor splice site module for biological and biotechnological applications SG11202106356QA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP18214415 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
SG11202106356QA true SG11202106356QA (en) 2021-07-29

Family

ID=64755152

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202106356QA SG11202106356QA (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

Family Cites Families (16)

* 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
US20020193580A1 (en) * 1995-12-15 2002-12-19 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
AU2014255665B2 (en) 2013-04-18 2018-08-02 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
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

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
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
SG11202106356QA (en) An optimized acceptor splice site module for biological and biotechnological applications
EP3345040A4 (en) DIFFERENTIAL REAR LIGHTS INDICATOR AND SYSTEM
EP3016147B8 (en) Solar cell module and method and device for repairing the same
EP3373058A4 (en) Lens driving device, and camera module and optical instrument comprising same
EP3321565A4 (en) BACKLIGHT MODULE AND DISPLAY DEVICE
EP3346325A4 (en) BACKLIGHT MODULE AND DISPLAY DEVICE
EP3358937A4 (en) CLOSED GROWTH SPACE AND AIR CONDITIONING SYSTEM
EP3567407A4 (en) BACKLIGHT MODULE, DISPLAY MODULE AND DISPLAY DEVICE
EP3396762A4 (en) FUEL CELL SYSTEM AND METHOD FOR CONTROLLING THIS SYSTEM
DK3306759T3 (da) Optisk fiber til forstærkning og laserindretning
DK3399580T3 (da) Brændstofcellesystem og fremgangsmåde til at drive et brændstofcellesystem
EP3607373A4 (en) OPTICAL TERMINATION AND SPLICE MODULE
EP3780299A4 (en) Laser module and laser device
EP3512017A4 (en) FUEL CELL SYSTEM AND METHOD FOR CONTROLLING THEREOF
EP3808833A4 (en) CELL CULTURE SYSTEM AND CELL CULTURE PROCESS
EP3808835A4 (en) CELL CULTURE SYSTEM AND CELL CULTURE PROCESS
EP3273838A4 (en) Light source optical system and light source device
EP3287690A4 (en) BACKLIGHT MODULE AND DISPLAY DEVICE
EP3835845A4 (en) OPTICAL MICROSCOPE DEVICE AND OPTICAL MICROSCOPE SYSTEM
EP3697889A4 (en) MULTI-AXIS MECHANICAL STIMULATION SYSTEMS AND INCUBATORS
EP3793739A4 (en) PORTABLE MICROFLUIDIC SYSTEM FOR BIOLOGICAL AND ANALYTICAL TESTING
EP3677954A4 (en) BACKLIGHT MODULE AND DISPLAY DEVICE
EP3641162A4 (en) METHOD AND DEVICE FOR COMPENSATING OPTICAL PERFORMANCE
EP3493919A4 (en) Multi-functional fluid dispensing system and associated methods for dispensing
HUE064293T2 (hu) Napelemmodul-rögzítõ- és tartórendszer