MA54958A - Modulation de l'activité de la protéine rep dans la production d'adn à extrémité fermée - Google Patents

Modulation de l'activité de la protéine rep dans la production d'adn à extrémité fermée

Info

Publication number
MA54958A
MA54958A MA054958A MA54958A MA54958A MA 54958 A MA54958 A MA 54958A MA 054958 A MA054958 A MA 054958A MA 54958 A MA54958 A MA 54958A MA 54958 A MA54958 A MA 54958A
Authority
MA
Morocco
Prior art keywords
modulation
closed
protein activity
rep protein
end dna
Prior art date
Application number
MA054958A
Other languages
English (en)
Inventor
Robert Michael Kotin
Ara Karl Malakian
Anna Ucher
Original Assignee
Generation Bio Co
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 Generation Bio Co filed Critical Generation Bio Co
Publication of MA54958A publication Critical patent/MA54958A/fr

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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
    • C12N15/79—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
    • 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
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6009—Vectors comprising as targeting moiety peptide derived from defined protein from viruses dsDNA viruses
    • C12N2810/6018—Adenoviridae
    • 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
    • C12N2820/00—Vectors comprising a special origin of replication system
    • C12N2820/002—Vectors comprising a special origin of replication system inducible or controllable
    • 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
    • C12N2820/00—Vectors comprising a special origin of replication system
    • C12N2820/60—Vectors comprising a special origin of replication system from viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
MA054958A 2019-02-15 2020-02-14 Modulation de l'activité de la protéine rep dans la production d'adn à extrémité fermée MA54958A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962806076P 2019-02-15 2019-02-15

Publications (1)

Publication Number Publication Date
MA54958A true MA54958A (fr) 2021-12-22

Family

ID=72045641

Family Applications (1)

Application Number Title Priority Date Filing Date
MA054958A MA54958A (fr) 2019-02-15 2020-02-14 Modulation de l'activité de la protéine rep dans la production d'adn à extrémité fermée

Country Status (11)

Country Link
US (1) US20220127625A1 (fr)
EP (1) EP3924491A4 (fr)
JP (2) JP7644014B2 (fr)
KR (1) KR20210127935A (fr)
CN (1) CN113454232B (fr)
AU (1) AU2020221312A1 (fr)
CA (1) CA3129321A1 (fr)
IL (1) IL285415A (fr)
MA (1) MA54958A (fr)
SG (1) SG11202106491VA (fr)
WO (1) WO2020168222A1 (fr)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI904068B (zh) 2017-08-09 2025-11-11 美商生物化學醫療公司 核酸分子及其用途
WO2020033863A1 (fr) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Molécules d'acide nucléique et leurs utilisations pour une thérapie génique non virale
CA3142932A1 (fr) * 2019-06-10 2020-12-17 Homology Medicines, Inc. Compositions de virus adeno-associes pour transfert de gene arsa et leurs procedes d'utilisation
EP4189098A1 (fr) 2020-07-27 2023-06-07 Anjarium Biosciences AG Compositions de molécules d'adn, leurs procédés de fabrication et leurs procédés d'utilisation
IL301196A (en) * 2020-09-16 2023-05-01 Generation Bio Co Closed-end DNA vectors and their uses for phenylalanine hydroxylase (pah) expression
EP4267197A1 (fr) * 2020-12-23 2023-11-01 Vivet Therapeutics Promoteurs inductibles par l'acide biliaire minimal pour thérapie génique
CA3213820A1 (fr) * 2021-03-16 2022-09-22 Wisconsin Alumni Research Foundation Therapie genique de l'insuline pour traiter le diabete
CA3229668A1 (fr) * 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Systeme d'expression de baculovirus
CN114703203B (zh) * 2022-02-11 2024-08-06 上海渤因生物科技有限公司 杆状病毒载体及其用途
KR102737146B1 (ko) * 2023-07-10 2024-12-09 대한민국 신규 발현 벡터 및 이의 용도
WO2025114932A1 (fr) * 2023-11-30 2025-06-05 Sanofi Purification de molécules d'adn à extrémité fermée
US12509711B1 (en) * 2024-08-09 2025-12-30 Martin Williams System and process for in vivo manufacturing nanostructure-ended double-stranded covalently-closed linear DNA, the resulting molecules and their uses

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2596292C (fr) * 1997-03-21 2012-05-22 Enzo Therapeutics, Inc. Vecteurs et vecteurs viraux et lignees de cellules d'encapsidation les propageant
WO2001032899A1 (fr) * 1999-10-29 2001-05-10 Takara Shuzo Co., Ltd. Methode de transfert de gene
KR101589259B1 (ko) * 2006-06-21 2016-02-01 유니큐어 아이피 비.브이. 곤충세포 내 ａａｖ의 생산에 유용한 ａａｖ-ｒｅｐ78의 번역을 위한 변형된 개시 코돈을 갖는 벡터
EP2545165B1 (fr) * 2010-03-11 2020-07-29 uniQure IP B.V. Séquences codant une protéine rep mutée destinées à être utilisées dans la production d'aav
EP2500434A1 (fr) * 2011-03-12 2012-09-19 Association Institut de Myologie Vecteurs AAV sans capside, compositions et procédés pour la production des vecteurs et la thérapie génique
KR102572449B1 (ko) * 2014-03-10 2023-08-31 유니큐어 아이피 비.브이. 곤충 세포에서 생산된 더욱 향상된 aav 벡터
EP3423110B1 (fr) * 2016-03-03 2021-08-11 University of Massachusetts Adn double hélice linéaire à extrémité fermée pour transfert de gène non viral
TWI904068B (zh) * 2017-08-09 2025-11-11 美商生物化學醫療公司 核酸分子及其用途

Also Published As

Publication number Publication date
KR20210127935A (ko) 2021-10-25
AU2020221312A1 (en) 2021-10-07
EP3924491A4 (fr) 2022-12-14
SG11202106491VA (en) 2021-07-29
JP2024083567A (ja) 2024-06-21
JP7644014B2 (ja) 2025-03-11
EP3924491A1 (fr) 2021-12-22
CN113454232A (zh) 2021-09-28
JP2022520803A (ja) 2022-04-01
CN113454232B (zh) 2024-10-29
US20220127625A1 (en) 2022-04-28
WO2020168222A1 (fr) 2020-08-20
CA3129321A1 (fr) 2020-08-20
IL285415A (en) 2021-09-30

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