BR112023024985A2 - Sistemas de edição de genes compreendendo uma crispr nuclease e usos dos mesmos - Google Patents

Sistemas de edição de genes compreendendo uma crispr nuclease e usos dos mesmos

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Publication number
BR112023024985A2
BR112023024985A2 BR112023024985A BR112023024985A BR112023024985A2 BR 112023024985 A2 BR112023024985 A2 BR 112023024985A2 BR 112023024985 A BR112023024985 A BR 112023024985A BR 112023024985 A BR112023024985 A BR 112023024985A BR 112023024985 A2 BR112023024985 A2 BR 112023024985A2
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Prior art keywords
crispr nuclease
nucleic acid
polypeptide
acid encoding
gene editing
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BR112023024985A
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English (en)
Inventor
A Scott David
Fu-Kai Hsieh
Michael Jakimo Noah
Pratyusha Hunnewell
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Arbor Biotechnologies Inc
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Application filed by Arbor Biotechnologies Inc filed Critical Arbor Biotechnologies Inc
Publication of BR112023024985A2 publication Critical patent/BR112023024985A2/pt

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Abstract

sistemas de edição de genes compreendendo uma crispr nuclease e usos dos mesmos. um sistema de edição de gene compreendendo: (a) um polipeptídeo de crispr nuclease tipo v ou um primeiro ácido nucleico codificando o polipeptídeo de crispr nuclease tipo v; (b) um polipeptídeo de transcriptase reversa (rt) ou um segundo ácido nucleico codificando o polipeptídeo de rt; (c) um rna guia (grna) ou um terceiro ácido nucleico codificando o grna, em que o grna compreende um ou mais sítios de ligação reconhecíveis pela crispr nuclease tipo v (sítios de ligação de crispr nuclease) e uma sequência espaçadora específica para uma sequência alvo dentro de um sítio genômico de interesse, a sequência alvo sendo adjacente a um motivo adjacente de protoespaçador (pam); e (d) um rna doador de transcrição reversa (rna doador de rt) ou um quarto ácido nucleico codificando o rna doador de rt, em que o rna doador de rt compreende um sítio de ligação de iniciador (pbs) e uma sequência modelo.
BR112023024985A 2021-06-01 2022-06-01 Sistemas de edição de genes compreendendo uma crispr nuclease e usos dos mesmos BR112023024985A2 (pt)

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US202163195621P 2021-06-01 2021-06-01
US202163236047P 2021-08-23 2021-08-23
US202163272937P 2021-10-28 2021-10-28
US202263299695P 2022-01-14 2022-01-14
PCT/US2022/031821 WO2022256440A2 (en) 2021-06-01 2022-06-01 Gene editing systems comprising a crispr nuclease and uses thereof

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US (2) US20240102007A1 (pt)
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JP (1) JP2024520528A (pt)
KR (1) KR20240031238A (pt)
AU (1) AU2022284804A1 (pt)
BR (1) BR112023024985A2 (pt)
CA (1) CA3222023A1 (pt)
IL (1) IL308806A (pt)
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