BR112013023185B8 - Molécula de ácido nucléico linear isolada, método para produzir uma molécula de ácido nucléico linear isolada, método in vitro para mediar a expressão do dna exógeno em uma célula de mamífero e método in vitro para efetuar a expressão do produto de gene dessejado ou oligonucleotídeo em uma célula - Google Patents

Molécula de ácido nucléico linear isolada, método para produzir uma molécula de ácido nucléico linear isolada, método in vitro para mediar a expressão do dna exógeno em uma célula de mamífero e método in vitro para efetuar a expressão do produto de gene dessejado ou oligonucleotídeo em uma célula

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BR112013023185B8
BR112013023185B8 BR112013023185A BR112013023185A BR112013023185B8 BR 112013023185 B8 BR112013023185 B8 BR 112013023185B8 BR 112013023185 A BR112013023185 A BR 112013023185A BR 112013023185 A BR112013023185 A BR 112013023185A BR 112013023185 B8 BR112013023185 B8 BR 112013023185B8
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nucleic acid
acid molecule
expression
linear nucleic
isolated linear
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BR112013023185A
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BR112013023185B1 (pt
BR112013023185A2 (pt
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Cyriaque Beley
Lina Li
Luis Garcia
Michael Kotin Robert
Thomas Voit
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Association Inst De Myologie
Centre Nat Rech Scient
Inserm Institut National De La Sante Et La Rech Medicale
Univ Pierre Et Marie Curie Paris 6
Us Department Of Health And Human Services Nat Institutes Of Health
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Application filed by Association Inst De Myologie, Centre Nat Rech Scient, Inserm Institut National De La Sante Et La Rech Medicale, Univ Pierre Et Marie Curie Paris 6, Us Department Of Health And Human Services Nat Institutes Of Health filed Critical Association Inst De Myologie
Publication of BR112013023185A2 publication Critical patent/BR112013023185A2/pt
Publication of BR112013023185B1 publication Critical patent/BR112013023185B1/pt
Publication of BR112013023185B8 publication Critical patent/BR112013023185B8/pt

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Abstract

vetores aav isentos de capsídío, composições e métodos para a produção de vetor e liberação de gene um molécula de ácido nucléico linear isolada que compreende nesta ordem: uma primeira repetição de terminal invertida (itr) de vírus adenoassociado (aav), uma sequência de nucleotídeo de interesse e uma segunda itr de avv, em que a dita molécula de ácido nucléico é destituída das sequências que codificam a proteína capsidica de aav. a , dita molécula de ácido nucléico pode ser aplicada a um hospedeiro em repetição sem evocar uma resposta imune. métodos para produzir e purificar esta molécula de ácido nucléico, e uso da mesma para propósitos terapêuticos também são fornecidos.
BR112013023185A 2011-03-11 2012-03-12 Molécula de ácido nucléico linear isolada, método para produzir uma molécula de ácido nucléico linear isolada, método in vitro para mediar a expressão do dna exógeno em uma célula de mamífero e método in vitro para efetuar a expressão do produto de gene dessejado ou oligonucleotídeo em uma célula BR112013023185B8 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201161452071P 2011-03-11 2011-03-11
US61/452,071 2011-03-11
EP11157986A EP2500434A1 (en) 2011-03-12 2011-03-12 Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
EP11157986.8 2011-03-12
PCT/EP2012/054303 WO2012123430A1 (en) 2011-03-11 2012-03-12 Capsid-free aav vectors, compositions, and methods for vector production and gene delivery

Publications (3)

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BR112013023185A2 BR112013023185A2 (pt) 2017-01-03
BR112013023185B1 BR112013023185B1 (pt) 2022-02-01
BR112013023185B8 true BR112013023185B8 (pt) 2022-03-22

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BR112013023185A BR112013023185B8 (pt) 2011-03-11 2012-03-12 Molécula de ácido nucléico linear isolada, método para produzir uma molécula de ácido nucléico linear isolada, método in vitro para mediar a expressão do dna exógeno em uma célula de mamífero e método in vitro para efetuar a expressão do produto de gene dessejado ou oligonucleotídeo em uma célula

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US (1) US9598703B2 (pt)
EP (3) EP2500434A1 (pt)
JP (6) JP2014513928A (pt)
KR (2) KR20140023310A (pt)
CN (1) CN103764831B (pt)
AU (1) AU2012228376B2 (pt)
BR (1) BR112013023185B8 (pt)
CA (1) CA2829518A1 (pt)
IL (1) IL228328B (pt)
WO (1) WO2012123430A1 (pt)

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US9598703B2 (en) 2017-03-21
KR20200116550A (ko) 2020-10-12
BR112013023185B1 (pt) 2022-02-01
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CA2829518A1 (en) 2012-09-20
WO2012123430A1 (en) 2012-09-20
CN103764831A (zh) 2014-04-30
EP2500434A1 (en) 2012-09-19
BR112013023185A2 (pt) 2017-01-03
KR20140023310A (ko) 2014-02-26
IL228328B (en) 2018-08-30
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JP2014513928A (ja) 2014-06-19
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