AR123777A1 - Constructos de ácido nucleico para transcripción de arn va - Google Patents

Constructos de ácido nucleico para transcripción de arn va

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Publication number
AR123777A1
AR123777A1 ARP210102827A ARP210102827A AR123777A1 AR 123777 A1 AR123777 A1 AR 123777A1 AR P210102827 A ARP210102827 A AR P210102827A AR P210102827 A ARP210102827 A AR P210102827A AR 123777 A1 AR123777 A1 AR 123777A1
Authority
AR
Argentina
Prior art keywords
adenoviral
open reading
nucleic acid
reading frame
rna
Prior art date
Application number
ARP210102827A
Other languages
English (en)
Inventor
Ulrich Goepfert
Simon Auslaender
Original Assignee
Hoffmann La Roche
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 Hoffmann La Roche filed Critical Hoffmann La Roche
Publication of AR123777A1 publication Critical patent/AR123777A1/es

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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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0681—Cells of the genital tract; Non-germinal cells from gonads
    • C12N5/0682—Cells of the female genital tract, e.g. endometrium; Non-germinal cells from ovaries, e.g. ovarian follicle cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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    • 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
    • C12N2750/14123—Virus like particles [VLP]
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    • 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
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    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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

Abstract

Reivindicación 1: Un ácido nucleico de ARN VA adenoviral que comprende, en dirección 5’ a 3’ un promotor de ARN U6 humano y una secuencia codificante de ARN VA adenoviral de SEQ ID Nº 38. Reivindicación 2: Un ácido nucleico de ARN VA adenoviral que comprende, en dirección 5’ a 3’ un promotor inducible y una secuencia codificante de ARN VA adenoviral de SEQ ID Nº 38. Reivindicación 3: Un ADN que comprende un ácido nucleico de ARN VA adenoviral de acuerdo con una cualquiera de las reivindicaciones 1 ó 2, y un elemento de ADN que comprende un marco de lectura abierto E1A y un marco de lectura abierto E1B; o un marco de lectura abierto E2A y un marco de lectura abierto E4 o E4orf6; o un marco de lectura abierto rep y un marco de lectura abierto cap. Reivindicación 4: Una célula de mamífero o de insecto que comprende un ácido nucleico de ARN VA adenoviral de acuerdo con una cualquiera de las reivindicaciones 1 a 2 o un ADN de acuerdo con la reivindicación 3. Reivindicación 5: Un método para producir partículas de virus adenoasociado recombinante que comprende: proporcionar una célula de mamífero, cultivada en suspensión, que comprende un casete de expresión transgénica interespaciado entre dos ITR de AAV; marcos de lectura abiertos que codifican las proteínas E1A, E1B, E2A, E4 o E4orf6 adenovirales; un ácido nucleico de ARN VA adenoviral de acuerdo con una cualquiera de las reivindicaciones 1 a 2; marcos de lectura abiertos que codifican proteínas Rep / Cap adenoasociadas; cultivar la célula de mamífero; y aislar las partículas de rAAV de la célula o el medio de cultivo y, de esta manera, producir partículas de rAAV.
ARP210102827A 2020-10-15 2021-10-13 Constructos de ácido nucleico para transcripción de arn va AR123777A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP20202010 2020-10-15

Publications (1)

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AR123777A1 true AR123777A1 (es) 2023-01-11

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ID=72964437

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ARP210102827A AR123777A1 (es) 2020-10-15 2021-10-13 Constructos de ácido nucleico para transcripción de arn va

Country Status (12)

Country Link
US (1) US20220135954A1 (es)
EP (1) EP4229205A1 (es)
JP (1) JP2023546116A (es)
KR (1) KR20230085929A (es)
CN (1) CN116406425A (es)
AR (1) AR123777A1 (es)
AU (1) AU2021360197A1 (es)
CA (1) CA3197730A1 (es)
IL (1) IL302046A (es)
MX (1) MX2023004052A (es)
TW (1) TW202223094A (es)
WO (1) WO2022079083A1 (es)

Family Cites Families (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US6093699A (en) 1987-07-09 2000-07-25 The University Of Manitoba Method for gene therapy involving suppression of an immune response
DK0452457T3 (da) 1989-11-03 1998-03-02 Univ Vanderbilt Fremgangsmåde til in vivo fjernelse af funktionelle fremmede gener
US5279833A (en) 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
WO1991018088A1 (en) 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
EP0557459B1 (en) 1990-11-13 1997-10-22 Immunex Corporation Bifunctional selectable fusion genes
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
IL105914A0 (en) 1992-06-04 1993-10-20 Univ California Methods and compositions for in vivo gene therapy
WO1994017810A1 (en) 1993-02-12 1994-08-18 The Wistar Institute Of Anatomy And Biology Recombinant cytomegalovirus vaccine
CA2160583A1 (en) 1993-04-16 1994-10-27 Stanley A. Plotkin Recombinant cytomegalovirus vaccine
US5670361A (en) 1993-05-17 1997-09-23 The Regents Of The University Of California HIV-specific ribozymes
EP0804590A1 (en) 1993-05-21 1997-11-05 Targeted Genetics Corporation Bifunctional selectable fusion genes based on the cytosine deaminase (cd) gene
US5998205A (en) 1994-11-28 1999-12-07 Genetic Therapy, Inc. Vectors for tissue-specific replication
WO1996026742A1 (en) 1995-02-28 1996-09-06 The Regents Of The University Of California Gene transfer-mediated angiogenesis therapy
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
EP1983057A3 (en) 1995-09-08 2009-01-07 Genzyme Corporation Improved AAV vectors for gene therapy
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
WO1997032481A1 (en) 1996-03-07 1997-09-12 The Regents Of The University Of California Helper-free, totally defective adenovirus for gene therapy
DE19955558C2 (de) 1999-11-18 2003-03-20 Stefan Kochanek Permanente Amniozyten-Zelllinie, ihre Herstellung und Verwendung zur Herstellung von Gentransfervektoren
AU2001257611A1 (en) 2000-04-28 2001-11-12 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
EP1546397A4 (en) 2002-09-27 2007-10-31 Cold Spring Harbor Lab CELL-BASED RNA INTERFERENCE AND METHODS AND COMPOSITIONS RELATING THERETO
FR2852021B1 (fr) * 2003-03-04 2007-12-07 Methodes et outils pour le criblage d'arn actifs in cellulo
EP2277996B1 (en) 2003-05-21 2014-09-03 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
DE102005054628A1 (de) 2005-11-16 2007-05-24 Cevec Pharmaceuticals Gmbh Verfahren zur Herstellung von permanenten humanen Zelllinien
ES2680915T3 (es) 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica
US20130058871A1 (en) 2011-07-28 2013-03-07 Howard Hughes Medical Institute Method and system for mapping synaptic connectivity using light microscopy
EP3795581A3 (en) 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
HUE054087T2 (hu) 2012-04-18 2021-09-28 Childrens Hospital Philadelphia Készítmények és eljárások nagy hatékonyságú géntranszferre AAV kapszidvariánsok alkalmazásával
PT3024498T (pt) 2013-07-22 2020-03-06 Childrens Hospital Philadelphia Variante aav e composições, métodos e usos para transferência genética em células, órgãos e tecidos
EP3039129B1 (en) * 2013-08-30 2018-06-27 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
EP3204050A4 (en) 2014-10-09 2018-04-04 The Regents of The University of California Targeted disruption of a csf1-dap12 pathway member gene for the treatment of neuropathic pain
AU2016362317B2 (en) 2015-12-01 2023-03-16 Spark Therapeutics, Inc. Scalable methods for producing recombinant Adeno-Associated Viral (AAV) vector in serum-free suspension cell culture system suitable for clinical use
JP2020507331A (ja) * 2017-02-17 2020-03-12 ロンザ リミテッドＬｏｎｚａ Ｌｉｍｉｔｅｄ アデノ随伴ウイルスを生産するための哺乳動物細胞
AU2018281306A1 (en) 2017-06-07 2020-01-16 Spark Therapeutics, Inc. Enhancing agents for improved cell transfection and/or rAAV vector production
PE20200737A1 (es) 2017-06-30 2020-07-23 Spark Therapeutics Inc Metodos de purificacion de columna de vector aav
US20210002669A1 (en) 2017-12-22 2021-01-07 Genentech, Inc. Targeted integration of nucleic acids
SG11202106217TA (en) * 2018-12-21 2021-07-29 Lonza Walkersville Inc Adeno-associated virus (aav) producer cell line and related methods

Also Published As

Publication number Publication date
JP2023546116A (ja) 2023-11-01
CN116406425A (zh) 2023-07-07
TW202223094A (zh) 2022-06-16
WO2022079083A1 (en) 2022-04-21
MX2023004052A (es) 2023-05-03
EP4229205A1 (en) 2023-08-23
AU2021360197A1 (en) 2023-05-18
US20220135954A1 (en) 2022-05-05
IL302046A (en) 2023-06-01
AU2021360197A9 (en) 2023-07-13
CA3197730A1 (en) 2022-04-21
KR20230085929A (ko) 2023-06-14

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