BR112018005620A2 - método para transferência de gene estável e de nível elevado em linfócitos - Google Patents

método para transferência de gene estável e de nível elevado em linfócitos

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Publication number
BR112018005620A2
BR112018005620A2 BR112018005620A BR112018005620A BR112018005620A2 BR 112018005620 A2 BR112018005620 A2 BR 112018005620A2 BR 112018005620 A BR112018005620 A BR 112018005620A BR 112018005620 A BR112018005620 A BR 112018005620A BR 112018005620 A2 BR112018005620 A2 BR 112018005620A2
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Brazil
Prior art keywords
lymphocytes
transposase
encoded
receptor
gene transfer
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BR112018005620A
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English (en)
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Hudecek Michael
Ivics Zoltan
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Univ Wuerzburg J Maximilians
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Publication of BR112018005620A2 publication Critical patent/BR112018005620A2/pt

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    • 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
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    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
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    • A61K39/46—Cellular immunotherapy
    • A61K39/461—Cellular immunotherapy characterised by the cell type used
    • A61K39/4611—T-cells, e.g. tumor infiltrating lymphocytes [TIL], lymphokine-activated killer cells [LAK] or regulatory T cells [Treg]
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    • A61K39/4631—Chimeric Antigen Receptors [CAR]
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    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
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Abstract

o método descrito aqui descreve uma nova tecnologia oferecendo eficiência não paralela, flexibilidade, utilidade e velocidade para integração estável de transgenes em linfócitos e outras células mamíferas. o novo método é baseado no uso de uma transposase codificada por mrna (por exemplo, sleeping beauty transposase) em combinação com um elemento transponível codificado por dna de minicírculo. o novo método permite taxas de transferência de gene maiores e é ao mesmo tempo menos tóxico do que o método convencional, que é o uso de transposase codificada por dna de plasmídeo em combinação com um elemento transponível codificado por dna de plasmídeo. as aplicações da invenção incluem, porém não estão limitadas à integração estável de um transgene codificando um receptor imune (por exemplo, um receptor de célula t ou um receptor de antígeno quimérico sintético) em linfócitos t humanos, com o receptor imune conferindo especificidade para uma molécula expressa por uma célula de tumor. o mrna de transposase e dna de minicírculo de transpóson podem ser introduzidos em linfócitos por métodos incluindo, porém não limitados à eletrotransferência tal como eletroporação e nucleofecção.
BR112018005620A 2015-09-22 2016-09-22 método para transferência de gene estável e de nível elevado em linfócitos BR112018005620A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP15002732 2015-09-22
EP16153490 2016-01-29
PCT/EP2016/072524 WO2017050884A1 (en) 2015-09-22 2016-09-22 A method for high level and stable gene transfer in lymphocytes

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BR112018005620A2 true BR112018005620A2 (pt) 2018-10-09

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Country Status (11)

Country Link
US (1) US20190169637A1 (pt)
EP (1) EP3352798A1 (pt)
JP (2) JP7142571B2 (pt)
KR (1) KR20180054718A (pt)
CN (1) CN108601849A (pt)
AU (1) AU2016325384B2 (pt)
BR (1) BR112018005620A2 (pt)
CA (1) CA2999608A1 (pt)
EA (1) EA201890772A1 (pt)
HK (1) HK1256068A1 (pt)
WO (1) WO2017050884A1 (pt)

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