BR112021018606A2 - Métodos e composições para editar sequências de nucleotídeos - Google Patents

Métodos e composições para editar sequências de nucleotídeos

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Publication number
BR112021018606A2
BR112021018606A2 BR112021018606A BR112021018606A BR112021018606A2 BR 112021018606 A2 BR112021018606 A2 BR 112021018606A2 BR 112021018606 A BR112021018606 A BR 112021018606A BR 112021018606 A BR112021018606 A BR 112021018606A BR 112021018606 A2 BR112021018606 A2 BR 112021018606A2
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Brazil
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methods
editing
prime editing
rna
compositions
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BR112021018606A
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English (en)
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Vito Anzalone Andrew
R Liu David
James Nelson
Peyton Randolph
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Harvard College
Broad Inst Inc
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Application filed by Harvard College, Broad Inst Inc filed Critical Harvard College
Publication of BR112021018606A2 publication Critical patent/BR112021018606A2/pt

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Abstract

métodos e composições para editar sequências de nucleotídeos. a presente revelação provê composições e métodos para realizar edição principal de uma molécula de dna alvo (por exemplo, (p.e.) um genoma) que permite a incorporação de uma alteração de nucleotídeo e/ou mutagênese direcionada. a alteração de nucleotídeo pode incluir uma alteração de nucleotídeo único (p.e., qualquer transição ou qualquer transversão), uma inserção de um ou mais nucleotídeos ou uma deleção de um ou mais nucleotídeos. mais em particular, a revelação provê proteínas de fusão compreendendo proteínas de ligação de dna programável de ácido nucleico (napdnabp) e uma polimerase (p.e., transcriptase reversa), que é guiada até uma sequência de dna específica por um rna de guia modificado, chamado pegrna. o pegrna foi alterado (em relação a um rna de guia padrão) para compreender uma parte estendida que provê uma sequência de gabarito de síntese de dna que codifica um flap de dna de fita única, que é homólogo a uma fita da sequência de dna endógena direcionada a ser editada, porém que contém uma ou mais alterações de nucleotídeo pretendidas e que, após síntese pela polimerase (p.e., transcriptase reversa), se torna incorporada na molécula de dna alvo. são também revelados métodos que impulsionam a edição principal, incluindo tratar doenças de contração de repetição de trinucleotídeo, instalar tags de peptídeo direcionado, tratar doença de príon através da instalação de mutações de proteção, manipular genes de codificação de rna para a instalação de tags de rna para controlar a função e expressão de rna, utilizar edição principal para construir bibliotecas sofisticadas de genes, utilizar edição principal para inserir imunoepítopos em proteínas, usar a edição principal para inserir domínios de dimerização induzíveis nos alvos de proteína, e métodos de aplicação, entre outros.
BR112021018606A 2019-03-19 2020-03-19 Métodos e composições para editar sequências de nucleotídeos BR112021018606A2 (pt)

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US201962820813P 2019-03-19 2019-03-19
US201962858958P 2019-06-07 2019-06-07
US201962922654P 2019-08-21 2019-08-21
US201962889996P 2019-08-21 2019-08-21
US201962973558P 2019-10-10 2019-10-10
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