BR112016025519A2 - engenharia genética in vivo com vetores de adenovírus - Google Patents

engenharia genética in vivo com vetores de adenovírus

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Publication number
BR112016025519A2
BR112016025519A2 BR112016025519A BR112016025519A BR112016025519A2 BR 112016025519 A2 BR112016025519 A2 BR 112016025519A2 BR 112016025519 A BR112016025519 A BR 112016025519A BR 112016025519 A BR112016025519 A BR 112016025519A BR 112016025519 A2 BR112016025519 A2 BR 112016025519A2
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Brazil
Prior art keywords
genetic engineering
adenovirus vectors
vivo genetic
helper
expression cassettes
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BR112016025519A
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English (en)
Inventor
Lieber Andre
Saydaminova Kamola
Richter Maximilian
Papayannopoulou Thalia
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Univ Washington
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Publication date
Application filed by Univ Washington filed Critical Univ Washington
Publication of BR112016025519A2 publication Critical patent/BR112016025519A2/pt

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    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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Abstract

a presente invenção fornece cassetes de expressão de ácidos nucleicos recombinantes e adenovírus dependente de auxiliar, onde os cassetes de expressão utilizam um sistema de mirna com base para controlar a expressão de nucleases em células produtoras de adenovírus dependentes de auxiliar, permitindo assim a produção e utilização para em edição de gene in vivo em células cd34 +.
BR112016025519A 2014-05-01 2015-05-01 engenharia genética in vivo com vetores de adenovírus BR112016025519A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201461987340P 2014-05-01 2014-05-01
PCT/US2015/028789 WO2015168547A2 (en) 2014-05-01 2015-05-01 In vivo gene engineering with adenoviral vectors

Publications (1)

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BR112016025519A2 true BR112016025519A2 (pt) 2018-01-16

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ID=54359500

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BR112016025519A BR112016025519A2 (pt) 2014-05-01 2015-05-01 engenharia genética in vivo com vetores de adenovírus

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US (1) US20170037431A1 (pt)
EP (1) EP3137120A4 (pt)
JP (1) JP2017514476A (pt)
CN (1) CN107405411A (pt)
BR (1) BR112016025519A2 (pt)
CA (1) CA2947466A1 (pt)
WO (1) WO2015168547A2 (pt)

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