MX2020008967A - Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma. - Google Patents

Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma.

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Publication number
MX2020008967A
MX2020008967A MX2020008967A MX2020008967A MX2020008967A MX 2020008967 A MX2020008967 A MX 2020008967A MX 2020008967 A MX2020008967 A MX 2020008967A MX 2020008967 A MX2020008967 A MX 2020008967A MX 2020008967 A MX2020008967 A MX 2020008967A
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MX
Mexico
Prior art keywords
delivery
compositions
methods
crispr
systems
Prior art date
Application number
MX2020008967A
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English (en)
Inventor
Le Cong
Matthias Heidenreich
Randall Jeffrey Platt
Lukasz Swiech
Feng Zhang
Cox David Benjamin Turitz
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Broad Inst Inc
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Publication date
Application filed by Broad Inst Inc filed Critical Broad Inst Inc
Publication of MX2020008967A publication Critical patent/MX2020008967A/es

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    • C12N9/22—Ribonucleases RNAses, DNAses
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    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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    • C12N15/1034—Isolating an individual clone by screening libraries
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/105—Murine
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2267/00—Animals characterised by purpose
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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Abstract

La invención proporciona el suministro, modificación y optimización de sistemas, métodos y composiciones para la manipulación de secuencias y/o actividades de secuencias diana. Se proporcionan sistemas de suministro y tejidos u órganos que se escogen como sitios diana para el suministro. También se proporcionan vectores y sistemas vectoriales, algunos de los cuales codifican uno o más componentes de un complejo CRISPR, así como también métodos para el diseño y uso de tales vectores. También se proporcionan métodos para dirigir la formación del complejo CRISPR en células eucariotas para garantizar una mejor especificidad en lo que se refiere al reconocimiento de la diana y para evitar toxicidad y para editar o modificar un sitio diana en un locus genómico de interés con el fin de alterar o mejorar el estado de una enfermedad o afección.
MX2020008967A 2013-12-12 2016-06-06 Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma. MX2020008967A (es)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201361915107P 2013-12-12 2013-12-12
US201361915176P 2013-12-12 2013-12-12
US201361915145P 2013-12-12 2013-12-12
US201361915215P 2013-12-12 2013-12-12
US201361915192P 2013-12-12 2013-12-12
US201361915153P 2013-12-12 2013-12-12
US201361915148P 2013-12-12 2013-12-12

Publications (1)

Publication Number Publication Date
MX2020008967A true MX2020008967A (es) 2020-11-09

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MX2016007328A MX2016007328A (es) 2013-12-12 2014-12-12 Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma.
MX2020008967A MX2020008967A (es) 2013-12-12 2016-06-06 Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma.

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MX2016007328A MX2016007328A (es) 2013-12-12 2014-12-12 Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma.

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US (2) US11407985B2 (es)
EP (2) EP3653229A1 (es)
JP (3) JP7103750B2 (es)
KR (1) KR20160089527A (es)
CN (2) CN111206032B (es)
AU (3) AU2014361781B2 (es)
BR (1) BR112016013201B1 (es)
CA (1) CA2932478A1 (es)
DK (1) DK3079725T3 (es)
ES (1) ES2765481T3 (es)
IL (2) IL246115B (es)
MX (2) MX2016007328A (es)
SG (1) SG10201804976YA (es)
WO (1) WO2015089462A1 (es)

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