CN119677849A - 用于足细胞特异性表达的核酸构建体和载体 - Google Patents

用于足细胞特异性表达的核酸构建体和载体 Download PDF

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Publication number
CN119677849A
CN119677849A CN202380051659.7A CN202380051659A CN119677849A CN 119677849 A CN119677849 A CN 119677849A CN 202380051659 A CN202380051659 A CN 202380051659A CN 119677849 A CN119677849 A CN 119677849A
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China
Prior art keywords
sequence
gene
promoter
nucleotide sequence
seq
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Pending
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CN202380051659.7A
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English (en)
Chinese (zh)
Inventor
A·梅塞格尔·纳瓦罗
M·西庸·罗德里格斯
J·P·阿雷瓦洛·巴蒂斯塔
G·费尔南德斯·米兰达
M·拉米斯·卡斯特托特
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Nineveh Treatment Co ltd
Waldenbrunn University Foundation Hospital Dresca Institute
Universitat Autonoma de Barcelona UAB
Institut de Ciencies Fotoniques ICFO
Original Assignee
Nineveh Treatment Co ltd
Waldenbrunn University Foundation Hospital Dresca Institute
Universitat Autonoma de Barcelona UAB
Institut de Ciencies Fotoniques ICFO
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Publication date
Application filed by Nineveh Treatment Co ltd, Waldenbrunn University Foundation Hospital Dresca Institute, Universitat Autonoma de Barcelona UAB, Institut de Ciencies Fotoniques ICFO filed Critical Nineveh Treatment Co ltd
Publication of CN119677849A publication Critical patent/CN119677849A/zh
Pending legal-status Critical Current

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    • 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
    • C12N15/67—General methods for enhancing the expression
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/36—Vector systems having a special element relevant for transcription being a transcription termination element
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Urology & Nephrology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202380051659.7A 2022-05-02 2023-04-28 用于足细胞特异性表达的核酸构建体和载体 Pending CN119677849A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP22382421.0A EP4273243A1 (en) 2022-05-02 2022-05-02 Nucleic acid constructs and vectors for podocyte specific expression
EP22382421.0 2022-05-02
PCT/EP2023/061388 WO2023213738A1 (en) 2022-05-02 2023-04-28 Nucleic acid constructs and vectors for podocyte specific expression

Publications (1)

Publication Number Publication Date
CN119677849A true CN119677849A (zh) 2025-03-21

Family

ID=82218428

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202380051659.7A Pending CN119677849A (zh) 2022-05-02 2023-04-28 用于足细胞特异性表达的核酸构建体和载体

Country Status (8)

Country Link
US (1) US20250288696A1 (enExample)
EP (2) EP4273243A1 (enExample)
JP (1) JP2025515979A (enExample)
KR (1) KR20250006979A (enExample)
CN (1) CN119677849A (enExample)
AU (1) AU2023266069A1 (enExample)
CA (1) CA3256851A1 (enExample)
WO (1) WO2023213738A1 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201900702D0 (en) * 2019-01-18 2019-03-06 Univ Bristol Therapy
GB202009039D0 (en) 2020-06-15 2020-07-29 Univ Bristol Therapy
GB202304841D0 (en) * 2023-03-31 2023-05-17 Purespring Therapeutics Ltd Promoter
GB202309909D0 (en) 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Regulatory elements
GB202309913D0 (en) * 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Polynucleotides, avv vectors, and pharmaceutical compositions for treating nephrotic syndrome
GB202309908D0 (en) * 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Polynucleotides, aav vectors, and pharmaceutical compositions for treating complement-mediated kidney diseases
GB202309914D0 (en) 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Promoters
WO2025168940A1 (en) 2024-02-07 2025-08-14 Purespring Therapeutics Limited Vectors
GB202407863D0 (en) 2024-06-03 2024-07-17 Purespring Therapeutics Ltd Methods
WO2026024176A1 (en) * 2024-07-22 2026-01-29 The Trustees Of The Columbia University In The City Of New York Synthetic reporters for multiplexed detection of transcription factor activity
GB202414499D0 (en) 2024-10-02 2024-11-13 Purespring Therapeutics Ltd Methods

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017153606A1 (en) 2016-03-11 2017-09-14 Fundació Institut De Recerca Biomèdica (Irb Barcelona) Nucleic acid constructs and vectors for oncoselective expression of a transgene
GB201900702D0 (en) 2019-01-18 2019-03-06 Univ Bristol Therapy
EP3914719A4 (en) * 2019-01-24 2023-04-05 Valorisation-HSJ, Limited Partnership Cell-specific transcriptional regulatory sequences and uses thereof
GB202003618D0 (en) 2020-03-12 2020-04-29 Univ Bristol Gene Therapy

Also Published As

Publication number Publication date
KR20250006979A (ko) 2025-01-13
WO2023213738A1 (en) 2023-11-09
AU2023266069A1 (en) 2024-12-19
CA3256851A1 (en) 2023-11-09
EP4519433A1 (en) 2025-03-12
US20250288696A1 (en) 2025-09-18
EP4273243A1 (en) 2023-11-08
JP2025515979A (ja) 2025-05-22

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