BR112015026422A2 - sistema de construto duplo para expressar a sequência de codificação de um gene de interesse em uma célula hospedeira, sistema de vetor viral duplo, célula hospedeira, composição farmacêutica, método para tratamento e/ou prevenção de uma patologia ou doença, ácido nucleico e método para induzir a recombinação genética - Google Patents

sistema de construto duplo para expressar a sequência de codificação de um gene de interesse em uma célula hospedeira, sistema de vetor viral duplo, célula hospedeira, composição farmacêutica, método para tratamento e/ou prevenção de uma patologia ou doença, ácido nucleico e método para induzir a recombinação genética

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Publication number
BR112015026422A2
BR112015026422A2 BR112015026422A BR112015026422A BR112015026422A2 BR 112015026422 A2 BR112015026422 A2 BR 112015026422A2 BR 112015026422 A BR112015026422 A BR 112015026422A BR 112015026422 A BR112015026422 A BR 112015026422A BR 112015026422 A2 BR112015026422 A2 BR 112015026422A2
Authority
BR
Brazil
Prior art keywords
host cell
dual
expressing
gene
interest
Prior art date
Application number
BR112015026422A
Other languages
English (en)
Other versions
BR112015026422B1 (pt
Inventor
Auricchio Alberto
Trapani Ivana
Colella Pasqualina
Original Assignee
Fond Telethon
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 Fond Telethon filed Critical Fond Telethon
Publication of BR112015026422A2 publication Critical patent/BR112015026422A2/pt
Publication of BR112015026422B1 publication Critical patent/BR112015026422B1/pt

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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

sistema de construto duplo para expressar a sequência de codificação de um gene de interesse em uma célula hospedeira, sistema de vetor viral duplo, célula hospedeira, composição farmacêutica, método para tratamento e/ou prevenção de uma patologia ou doença, ácido nucleico e método para induzir a recombinação genética abstract the present invention relates to constructs, vectors, relative host cells and pharmaceutical compositions which allow an effective gene therapy, in particular of genes larger than 5kb.
BR112015026422-0A 2013-04-18 2014-04-18 Sistema de construto duplo para expressar a sequência de codificação de um gene de interesse em uma célula hospedeira, sistema de vetor viral vírus adenoassociado (aav) duplo, célula hospedeira microbiana, composição farmacêutica e método in vitro para induzir a recombinação genética BR112015026422B1 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361813342P 2013-04-18 2013-04-18
US61/813,342 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
BR112015026422A2 true BR112015026422A2 (pt) 2017-11-14
BR112015026422B1 BR112015026422B1 (pt) 2023-05-23

Family

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Also Published As

Publication number Publication date
CA2909733A1 (en) 2014-10-23
EP2986635A1 (en) 2016-02-24
WO2014170480A1 (en) 2014-10-23
EA034575B1 (ru) 2020-02-21
US10494645B2 (en) 2019-12-03
AU2014255665A1 (en) 2015-11-12
CN105408352A (zh) 2016-03-16
JP2016516424A (ja) 2016-06-09
MX365711B (es) 2019-06-11
JP6453307B2 (ja) 2019-01-16
CA2909733C (en) 2023-10-17
MX2015014423A (es) 2016-07-28
EP2986635B1 (en) 2018-10-03
DK2986635T3 (en) 2019-01-28
ES2704677T3 (es) 2019-03-19
US20160076054A1 (en) 2016-03-17
CN105408352B (zh) 2020-01-14
EA201591992A1 (ru) 2016-04-29
AU2014255665B2 (en) 2018-08-02

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Legal Events

Date Code Title Description
B06F Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette]
B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]

Free format text: DE ACORDO COM O ARTIGO 229-C DA LEI NAO 10196/2001, QUE MODIFICOU A LEI NAO 9279/96, A CONCESSAO DA PATENTE ESTA CONDICIONADA A ANUAANCIA PRA VIA DA ANVISA. CONSIDERANDO A APROVAA AO DOS TERMOS DO PARECER NAO 337/PGF/EA/2010, BEM COMO A PORTARIA INTERMINISTERIAL NAO 1065 DE 24/05/2012, ENCAMINHA-SE O PRESENTE PEDIDO PARA AS PROVIDAANCIAS CABA-VEIS.

B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
B350 Update of information on the portal [chapter 15.35 patent gazette]
B07A Application suspended after technical examination (opinion) [chapter 7.1 patent gazette]
B09A Decision: intention to grant [chapter 9.1 patent gazette]
B16A Patent or certificate of addition of invention granted [chapter 16.1 patent gazette]

Free format text: PRAZO DE VALIDADE: 20 (VINTE) ANOS CONTADOS A PARTIR DE 18/04/2014, OBSERVADAS AS CONDICOES LEGAIS