BR112022008861A2 - Construto de ácido nucleico, vetor, nanopartícula lipídica, célula, composição para uso na expressão de retinosquisina, método para integrar uma sequência de codificação, e, método para modificar um gene rs1 - Google Patents

Construto de ácido nucleico, vetor, nanopartícula lipídica, célula, composição para uso na expressão de retinosquisina, método para integrar uma sequência de codificação, e, método para modificar um gene rs1

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BR112022008861A2
BR112022008861A2 BR112022008861A BR112022008861A BR112022008861A2 BR 112022008861 A2 BR112022008861 A2 BR 112022008861A2 BR 112022008861 A BR112022008861 A BR 112022008861A BR 112022008861 A BR112022008861 A BR 112022008861A BR 112022008861 A2 BR112022008861 A2 BR 112022008861A2
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nucleic acid
retinoschisin
cell
coding sequence
integrating
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BR112022008861A
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English (en)
Inventor
Hunt Charleen
Liu Yang
Gong Guochun
Romano Carmelo
Zambrowicz Brian
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Regeneron Pharma
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Publication of BR112022008861A2 publication Critical patent/BR112022008861A2/pt

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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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Abstract

CONSTRUTO DE ÁCIDO NUCLEICO, VETOR, NANOPARTÍCULA LIPÍDICA, CÉLULA, COMPOSIÇÃO PARA USO NA EXPRESSÃO DE RETINOSQUISINA, MÉTODO PARA INTEGRAR UMA SEQUÊNCIA DE CODIFICAÇÃO, E, MÉTODO PARA MODIFICAR UM GENE RS1. Trata se de construtos e composições de ácido nucleico que permitem a inserção e/ou expressão de uma sequência de codificação de retinosquisina. São fornecidos agentes de nuclease direcionados aos loci de RS1. São fornecidos, também, composições e métodos de uso de tais construtos para integração em um locus alvo genômico e/ou expressão em uma célula. São fornecidos, também, métodos de tratamento de retinosquise juvenil ligada ao X que usam os construtos e composições de ácido nucleico.
BR112022008861A 2019-11-08 2020-11-07 Construto de ácido nucleico, vetor, nanopartícula lipídica, célula, composição para uso na expressão de retinosquisina, método para integrar uma sequência de codificação, e, método para modificar um gene rs1 BR112022008861A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962932608P 2019-11-08 2019-11-08
PCT/US2020/059568 WO2021092513A1 (en) 2019-11-08 2020-11-07 Crispr and aav strategies for x-linked juvenile retinoschisis therapy

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BR112022008861A2 true BR112022008861A2 (pt) 2022-08-23

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US (1) US20230001019A1 (pt)
EP (1) EP4054651A1 (pt)
JP (1) JP2022553573A (pt)
KR (1) KR20220097414A (pt)
CN (1) CN114746125A (pt)
AU (1) AU2020379046A1 (pt)
BR (1) BR112022008861A2 (pt)
CA (1) CA3156277A1 (pt)
IL (1) IL292605A (pt)
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WO (1) WO2021092513A1 (pt)

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CA3156277A1 (en) 2021-05-14
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