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MX2018015629A - Sistema de vector viral adeno-asociado traslapado doble para la expresion de abc4a. - Google Patents

Sistema de vector viral adeno-asociado traslapado doble para la expresion de abc4a.

Info

Publication number
MX2018015629A
MX2018015629A MX2018015629A MX2018015629A MX2018015629A MX 2018015629 A MX2018015629 A MX 2018015629A MX 2018015629 A MX2018015629 A MX 2018015629A MX 2018015629 A MX2018015629 A MX 2018015629A MX 2018015629 A MX2018015629 A MX 2018015629A
Authority
MX
Mexico
Prior art keywords
nucleic acid
acid sequence
sequence
vector system
expressing
Prior art date
Application number
MX2018015629A
Other languages
English (en)
Inventor
Maclaren Robert
Mcclemments Michelle
Original Assignee
Univ Oxford Innovation Ltd
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
Priority claimed from GBGB1610448.1A external-priority patent/GB201610448D0/en
Priority claimed from GBGB1707261.2A external-priority patent/GB201707261D0/en
Application filed by Univ Oxford Innovation Ltd filed Critical Univ Oxford Innovation Ltd
Publication of MX2018015629A publication Critical patent/MX2018015629A/es

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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
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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
    • C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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  • Biomedical Technology (AREA)
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  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

La presente invención proporciona un sistema de vectores de virus adeno-asociados (AAV) para la expresión de una proteína de ABCA4 humana en una célula objetivo, en donde el sistema de vectores de AAV comprende un primer vector de AAV, el cual comprende una primera secuencia de ácido nucleico, y un segundo vector de AAV, el cual comprende una segunda secuencia de ácido nucleico; en donde la primera secuencia de ácido nucleico comprende una porción de extremo 5´ de una secuencia de codificación (CDS) de ABCA4, y la segunda secuencia de ácido nucleico comprende una porción de extremo 3´ de una CDS de ABCA4, y la porción de extremo 5´ y la porción de extremo 3´ abarcan juntas la CDS de ABCA4 entera; en donde la primera secuencia de ácido nucleico comprende una secuencia de nucleótidos contiguos que corresponden a los nucleótidos 105 a 3597 de la SEQ ID NO: 1; en donde la segunda secuencia de ácido nucleico comprende una secuencia de nucleótidos contiguos que corresponden a los nucleótidos 3806 a 6926 de la SEQ ID NO: 1; en donde cada una de la primera secuencia de ácido nucleico y la segunda secuencia de ácido nucleico comprende una región de la secuencia traslapada con la otra; y en donde la región de la secuencia traslapada comprende por lo menos aproximadamente 20 nucleótidos contiguos de una secuencia de ácido nucleico correspondientes a los nucleótidos 3598 a 3805 de la SEQ ID NO: 1. También se proporcionan los usos de los sistemas de vectores de AAV en la prevención o el tratamiento de enfermedades.
MX2018015629A 2016-06-15 2017-06-14 Sistema de vector viral adeno-asociado traslapado doble para la expresion de abc4a. MX2018015629A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1610448.1A GB201610448D0 (en) 2016-06-15 2016-06-15 Adeno-associated viral vector system
GBGB1707261.2A GB201707261D0 (en) 2017-05-05 2017-05-05 Adeno-associated viral vector system
PCT/GB2017/051741 WO2017216560A1 (en) 2016-06-15 2017-06-14 Dual overlapping adeno-associated viral vector system for expressing abc4a

Publications (1)

Publication Number Publication Date
MX2018015629A true MX2018015629A (es) 2019-09-26

Family

ID=59101505

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018015629A MX2018015629A (es) 2016-06-15 2017-06-14 Sistema de vector viral adeno-asociado traslapado doble para la expresion de abc4a.

Country Status (13)

Country Link
US (1) US20190309326A1 (es)
EP (1) EP3472328A1 (es)
JP (1) JP2019523648A (es)
KR (1) KR20190020745A (es)
CN (1) CN109642242A (es)
AU (1) AU2017286623A1 (es)
BR (1) BR112018075855A2 (es)
CA (1) CA3025445A1 (es)
IL (1) IL263523A (es)
MX (1) MX2018015629A (es)
RU (1) RU2765826C2 (es)
SG (1) SG11201811244SA (es)
WO (1) WO2017216560A1 (es)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA034575B1 (ru) 2013-04-18 2020-02-21 Фондацьоне Телетон Эффективная доставка больших генов посредством двойных aav векторов
AU2018261769B2 (en) 2017-05-05 2024-03-14 The Regents Of The University Of California Compositions and methods for expressing Otoferlin
JP2021520231A (ja) * 2018-04-05 2021-08-19 オックスフォード ユニバーシティ イノベーション リミテッドＯｘｆｏｒｄ Ｕｎｉｖｅｒｓｉｔｙ Ｉｎｎｏｖａｔｉｏｎ Ｌｉｍｉｔｅｄ シュタルガルト病の処置のための組成物及び方法
EP3867387A2 (en) * 2018-10-15 2021-08-25 Fondazione Telethon Intein proteins and uses thereof
GB201817469D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy for retinal disease
WO2020093018A1 (en) 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS
WO2021015997A1 (en) * 2019-07-15 2021-01-28 President And Fellows Of Harvard College Methods and compositions for gene delivery
EP4126071A4 (en) * 2020-04-01 2024-05-08 University Of Florida Research Foundation, Incorporated DUAL AAV-MYO7A VECTORS WITH IMPROVED SAFETY FOR THE TREATMENT OF USH1B
WO2022036318A1 (en) * 2020-08-14 2022-02-17 Case Western Reserve University Plasmid vectors and nanoparticles for treating ocular disorders
EP4334447A1 (en) * 2021-05-07 2024-03-13 UCL Business Ltd Abca4 genome editing
US20240425876A1 (en) * 2022-02-25 2024-12-26 Chigenovo Co., Ltd. Expression cassette combinations and uses thereof
WO2024257061A1 (en) * 2023-06-16 2024-12-19 Arkasubhra Ghosh A hybrid dual aav vector system with splice enhancer elements for expression of large genes

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI1945779T1 (sl) * 2005-10-20 2013-07-31 Uniqure Ip B.V. Izboljĺ ani aav vektorji proizvedeni v celicah insekta
US20090214478A1 (en) * 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
US9393299B2 (en) * 2009-08-07 2016-07-19 Transgene S.A. Composition for treating HBV infection
WO2013075008A1 (en) * 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
EA034575B1 (ru) * 2013-04-18 2020-02-21 Фондацьоне Телетон Эффективная доставка больших генов посредством двойных aav векторов

Also Published As

Publication number Publication date
WO2017216560A1 (en) 2017-12-21
US20190309326A1 (en) 2019-10-10
RU2019100525A3 (es) 2020-07-15
JP2019523648A (ja) 2019-08-29
BR112018075855A2 (pt) 2019-04-02
IL263523A (en) 2019-01-31
EP3472328A1 (en) 2019-04-24
KR20190020745A (ko) 2019-03-04
CA3025445A1 (en) 2017-12-21
CN109642242A (zh) 2019-04-16
RU2019100525A (ru) 2020-07-15
SG11201811244SA (en) 2019-01-30
AU2017286623A1 (en) 2018-12-20
RU2765826C2 (ru) 2022-02-03

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