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MX2016007326A - Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos. - Google Patents

Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.

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Publication number
MX2016007326A
MX2016007326A MX2016007326A MX2016007326A MX2016007326A MX 2016007326 A MX2016007326 A MX 2016007326A MX 2016007326 A MX2016007326 A MX 2016007326A MX 2016007326 A MX2016007326 A MX 2016007326A MX 2016007326 A MX2016007326 A MX 2016007326A
Authority
MX
Mexico
Prior art keywords
methods
nucleotide repeat
repeat disorders
compositions
delivery
Prior art date
Application number
MX2016007326A
Other languages
English (en)
Inventor
Zhang Feng
Rodriguez Edgardo
Davidson Beverly
Yu LIN Chie-
Original Assignee
Broad Inst Inc
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Filing date
Publication date
Application filed by Broad Inst Inc filed Critical Broad Inst Inc
Publication of MX2016007326A publication Critical patent/MX2016007326A/es

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Abstract

La invención proporciona el suministro, la manipulación y la optimización de sistemas, métodos y composiciones para la manipulación de secuencias y/o actividades de secuencias diana, especialmente para el uso que se refiere a trastornos de repetición de nucleótidos. Se proporcionan sistemas de suministro tejidos u órganos que se escogen como sitios diana para el suministro, especialmente para el uso relativo a trastornos de repetición de nucleótidos. También se proporcionan vectores y sistemas vectoriales, algunos de los cuales codifican uno o más componentes de un complejo o sistema CRISPR, especialmente para el uso relativo a trastornos de repetición de nucleótidos, así como a los métodos para el diseño y de tales. También se proporcionan métodos para dirigir la formación del sistema o complejo CRISPR en células eucariotas especialmente para el uso relativo a trastornos de repetición de nucleótidos incluyendo consideración de especificidad para el reconocimiento de la diana y para evitar toxicidad y editar o modificar un sitio diana en un locus genómico de interés con el fin de alternar o mejorar el estado de una enfermedad o afección.
MX2016007326A 2013-12-12 2014-12-12 Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos. MX2016007326A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
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

Publications (1)

Publication Number Publication Date
MX2016007326A true MX2016007326A (es) 2017-02-13

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
MX2016007325A MX2016007325A (es) 2013-12-12 2014-12-12 Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.
MX2016007326A MX2016007326A (es) 2013-12-12 2014-12-12 Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.

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MX2016007325A MX2016007325A (es) 2013-12-12 2014-12-12 Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.

Country Status (12)

Country Link
US (5) US10851357B2 (es)
EP (4) EP3080258A1 (es)
JP (2) JP6625055B2 (es)
KR (2) KR20160097338A (es)
CN (2) CN106029880A (es)
AU (2) AU2014362245A1 (es)
BR (2) BR112016013547A2 (es)
CA (2) CA2932472A1 (es)
IL (2) IL246118A0 (es)
MX (2) MX2016007325A (es)
SG (2) SG10201804973TA (es)
WO (2) WO2015089351A1 (es)

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