EA201591992A1 - Эффективная доставка больших генов посредством двойных aav векторов - Google Patents

Эффективная доставка больших генов посредством двойных aav векторов

Info

Publication number
EA201591992A1
EA201591992A1 EA201591992A EA201591992A EA201591992A1 EA 201591992 A1 EA201591992 A1 EA 201591992A1 EA 201591992 A EA201591992 A EA 201591992A EA 201591992 A EA201591992 A EA 201591992A EA 201591992 A1 EA201591992 A1 EA 201591992A1
Authority
EA
Eurasian Patent Office
Prior art keywords
aav vectors
effective delivery
genes
big
double aav
Prior art date
Application number
EA201591992A
Other languages
English (en)
Other versions
EA034575B1 (ru
Inventor
Альберто Ауриццио
Паскуалина Колелла
Ивана Трапани
Original Assignee
Фондацьоне Телетон
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 Фондацьоне Телетон filed Critical Фондацьоне Телетон
Publication of EA201591992A1 publication Critical patent/EA201591992A1/ru
Publication of EA034575B1 publication Critical patent/EA034575B1/ru

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

Abstract

Данное изобретение относится к конструктам, векторам, соответствующим клеткам-хозяина и фармацевтическим композициям, которые позволяют эффективную генную терапию, в частности генов больше чем 5 т.н.
EA201591992A 2013-04-18 2014-04-18 Эффективная доставка больших генов посредством двойных aav векторов EA034575B1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361813342P 2013-04-18 2013-04-18
PCT/EP2014/058000 WO2014170480A1 (en) 2013-04-18 2014-04-18 Effective delivery of large genes by dual aav vectors

Publications (2)

Publication Number Publication Date
EA201591992A1 true EA201591992A1 (ru) 2016-04-29
EA034575B1 EA034575B1 (ru) 2020-02-21

Family

ID=50678160

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201591992A EA034575B1 (ru) 2013-04-18 2014-04-18 Эффективная доставка больших генов посредством двойных aav векторов

Country Status (11)

Country Link
US (1) US10494645B2 (ru)
EP (1) EP2986635B1 (ru)
JP (1) JP6453307B2 (ru)
CN (1) CN105408352B (ru)
AU (1) AU2014255665B2 (ru)
CA (1) CA2909733C (ru)
DK (1) DK2986635T3 (ru)
EA (1) EA034575B1 (ru)
ES (1) ES2704677T3 (ru)
MX (1) MX365711B (ru)
WO (1) WO2014170480A1 (ru)

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Publication number Publication date
EP2986635A1 (en) 2016-02-24
JP6453307B2 (ja) 2019-01-16
AU2014255665B2 (en) 2018-08-02
CN105408352B (zh) 2020-01-14
DK2986635T3 (en) 2019-01-28
CN105408352A (zh) 2016-03-16
CA2909733A1 (en) 2014-10-23
JP2016516424A (ja) 2016-06-09
EP2986635B1 (en) 2018-10-03
US10494645B2 (en) 2019-12-03
EA034575B1 (ru) 2020-02-21
CA2909733C (en) 2023-10-17
AU2014255665A1 (en) 2015-11-12
MX2015014423A (es) 2016-07-28
US20160076054A1 (en) 2016-03-17
MX365711B (es) 2019-06-11
ES2704677T3 (es) 2019-03-19
BR112015026422A2 (pt) 2017-11-14
WO2014170480A1 (en) 2014-10-23

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