AU2023269540A1 - Use of transposases for improving transgene expression and nuclear localization - Google Patents

Use of transposases for improving transgene expression and nuclear localization Download PDF

Info

Publication number
AU2023269540A1
AU2023269540A1 AU2023269540A AU2023269540A AU2023269540A1 AU 2023269540 A1 AU2023269540 A1 AU 2023269540A1 AU 2023269540 A AU2023269540 A AU 2023269540A AU 2023269540 A AU2023269540 A AU 2023269540A AU 2023269540 A1 AU2023269540 A1 AU 2023269540A1
Authority
AU
Australia
Prior art keywords
transposase
protein
nucleic acid
amino acid
seq
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
AU2023269540A
Other languages
English (en)
Inventor
Marc GÜELL CARGOL
Maria PALLARÈS MASMITJÀ
Avencia SÁNCHEZ-MEJÍAS GARCIA
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.)
Integra Therapeutics
Universitat Pompeu Fabra UPF
Original Assignee
Integra Therapeutics
Universitat Pompeu Fabra UPF
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 Integra Therapeutics, Universitat Pompeu Fabra UPF filed Critical Integra Therapeutics
Publication of AU2023269540A1 publication Critical patent/AU2023269540A1/en
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C12N9/226Class 2 CAS enzyme complex, e.g. single CAS protein
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/12Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241Nucleotidyltransferases (2.7.7)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/90Vectors containing a transposable element

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2023269540A 2022-05-13 2023-05-12 Use of transposases for improving transgene expression and nuclear localization Pending AU2023269540A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263341619P 2022-05-13 2022-05-13
US63/341,619 2022-05-13
EP22191966 2022-08-24
EP22191966.5 2022-08-24
PCT/EP2023/062736 WO2023218021A1 (en) 2022-05-13 2023-05-12 Use of transposases for improving transgene expression and nuclear localization

Publications (1)

Publication Number Publication Date
AU2023269540A1 true AU2023269540A1 (en) 2024-11-07

Family

ID=86605260

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2023269540A Pending AU2023269540A1 (en) 2022-05-13 2023-05-12 Use of transposases for improving transgene expression and nuclear localization

Country Status (7)

Country Link
EP (1) EP4522735A1 (https=)
JP (1) JP2025518638A (https=)
KR (1) KR20250010025A (https=)
CN (1) CN119487184A (https=)
AU (1) AU2023269540A1 (https=)
CA (1) CA3253152A1 (https=)
WO (1) WO2023218021A1 (https=)

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110047635A1 (en) * 2006-08-28 2011-02-24 University of Hawail Methods and compositions for transposon-mediated transgenesis
CA3082450A1 (en) * 2017-11-21 2019-05-31 Crispr Therapeutics Ag Materials and methods for treatment of autosomal dominant retinitis pigmentosa
US12338453B2 (en) * 2018-12-17 2025-06-24 Consejo Superior De Investigaciones Científicas (Csic) Method for the introduction of genetic information in cell by site-specific integration system
WO2020243085A1 (en) 2019-05-24 2020-12-03 The Trustees Of Columbia University In The City Of New York Engineered cas-transposon system for programmable and site-directed dna transpositions
CN115968301A (zh) * 2020-04-20 2023-04-14 综合Dna技术公司 优化的蛋白融合和接头
MX2022015284A (es) * 2020-06-05 2023-01-19 Univ California Composiciones y metodos para la edicion del epigenoma.
CA3173696A1 (en) * 2020-07-17 2022-01-20 Volker Sandig Hyperactive transposons and transposases
AU2021403660A1 (en) 2020-12-16 2023-07-06 Universitat Pompeu Fabra Programmable transposases and uses thereof

Also Published As

Publication number Publication date
JP2025518638A (ja) 2025-06-18
CA3253152A1 (en) 2023-11-16
CN119487184A (zh) 2025-02-18
EP4522735A1 (en) 2025-03-19
WO2023218021A1 (en) 2023-11-16
KR20250010025A (ko) 2025-01-20

Similar Documents

Publication Publication Date Title
US20240167060A1 (en) Compositions and methods for genomic editing by insertion of donor polynucleotides
US10927383B2 (en) Cas9 mRNAs
AU2018338790B2 (en) Non-human animals comprising a humanized TTR locus and methods of use
CA3036926C (en) Modified stem cell memory t cells, methods of making and methods of using same
CN114007655A (zh) 用于细胞疗法的环状rna
KR102738651B1 (ko) 간에서 목적하는 단백질 발현하기 위한 플랫폼
KR20200032693A (ko) Cas-형질전환 마우스 배아 줄기 세포 및 마우스 및 이것의 용도
KR102915369B1 (ko) X-연관 연소 망막층간분리 치료법을 위한 crispr 및 aav 전략
US12448630B2 (en) CRISPR-Cas systems and uses thereof
WO2023193616A1 (zh) 单碱基编辑修复hba2基因突变的方法及其应用
CN110891419A (zh) 评价crispr/cas-诱导的与外源供体核酸的体内重组
CN118632622A (zh) 突变型肌纤蛋白疾病模型及其用途
EP3640334A1 (en) Genome editing system for repeat expansion mutation
AU2023269540A1 (en) Use of transposases for improving transgene expression and nuclear localization
US20250269057A1 (en) Therapeutic lama2 payload for treatment of congenital muscular dystrophy
US20220411826A1 (en) Co-opting regulatory bypass repair of genetic diseases
RU2833486C1 (ru) Стратегии crispr и aav для терапии х-сцепленного ювенильного ретиношизиса
RU2784927C1 (ru) Отличные от человека животные, включающие в себя гуманизированный ttr локус, и способы применения
WO2026059836A1 (en) Lipid nanoparticles and methods of use