BR112016013520A2 - Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos - Google Patents

Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos

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Publication number
BR112016013520A2
BR112016013520A2 BR112016013520A BR112016013520A BR112016013520A2 BR 112016013520 A2 BR112016013520 A2 BR 112016013520A2 BR 112016013520 A BR112016013520 A BR 112016013520A BR 112016013520 A BR112016013520 A BR 112016013520A BR 112016013520 A2 BR112016013520 A2 BR 112016013520A2
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Brazil
Prior art keywords
methods
nucleotide repeat
repeat disorders
compositions
crispr
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BR112016013520A
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English (en)
Inventor
Davidson Beverly
Lin Chie-Yu
Rodriguez Edgardo
Zhang Feng
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Broad Inst Inc
Massachusetts Inst Technology
Univ Iowa Res Found
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Application filed by Broad Inst Inc, Massachusetts Inst Technology, Univ Iowa Res Found filed Critical Broad Inst Inc
Publication of BR112016013520A2 publication Critical patent/BR112016013520A2/pt

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Abstract

COMPOSIÇÕES E MÉTODOS DE USO DE SISTEMAS CRISPR-CAS EM DISTÚRBIOS DE REPETIÇÕES DE NUCLEOTÍDEOS. A invenção proporciona a administração, manipulação e otimização de sistemas, métodos, e composições para manipulação de sequências e/ou atividades de sequências alvo, em especial para uso em distúrbios de repetições de nucleotídeos. São proporcionados sistemas de administração e tecidos ou órgãos que são visados como locais de administração, em especial para uso em distúrbios de repetições de nucleotídeos. Também são proporcionados vetores e sistemas de vetores, alguns dos quais codificam um ou mais componentes de um complexo ou sistema CRISPR, especialmente para uso em distúrbios de repetições de nucleotídeos, bem como métodos para o planejamento dos mesmos. Também são proporcionados métodos de direcionamento da formação do complexo ou sistema CRISPR em células eucarióticas especialmente para uso em distúrbios de repetições de nucleotídeos, incluindo a consideração da especificidade para reconhecimento de alvos e evitar a toxicidade e editar ou modificar um local alvo em um lócus genômico de interesse, para alterar o melhorar o estado de uma doença ou de um estado clínico.
BR112016013520A 2013-12-12 2014-12-12 Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos BR112016013520A2 (pt)

Applications Claiming Priority (4)

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US201361915150P 2013-12-12 2013-12-12
US201462010888P 2014-06-11 2014-06-11
US201462010879P 2014-06-11 2014-06-11
PCT/US2014/069902 WO2015089354A1 (en) 2013-12-12 2014-12-12 Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders

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BR112016013520A2 true BR112016013520A2 (pt) 2017-10-03

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BR112016013520A BR112016013520A2 (pt) 2013-12-12 2014-12-12 Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos

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US (5) US20160354487A1 (pt)
EP (4) EP3080257A1 (pt)
JP (2) JP6712948B2 (pt)
KR (2) KR20160097338A (pt)
CN (2) CN106029880A (pt)
AU (2) AU2014362245A1 (pt)
BR (2) BR112016013547A2 (pt)
CA (2) CA2932436A1 (pt)
IL (2) IL246118A0 (pt)
MX (2) MX2016007326A (pt)
SG (2) SG10201804973TA (pt)
WO (2) WO2015089354A1 (pt)

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