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BR112015031608A2 - Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos - Google Patents

Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos

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Publication number
BR112015031608A2
BR112015031608A2 BR112015031608A BR112015031608A BR112015031608A2 BR 112015031608 A2 BR112015031608 A2 BR 112015031608A2 BR 112015031608 A BR112015031608 A BR 112015031608A BR 112015031608 A BR112015031608 A BR 112015031608A BR 112015031608 A2 BR112015031608 A2 BR 112015031608A2
Authority
BR
Brazil
Prior art keywords
vectors
compositions
application
crispr
systems
Prior art date
Application number
BR112015031608A
Other languages
English (en)
Inventor
Zhang Feng
Cong Le
Ran Fei
Original Assignee
Massachusetts Inst Technology
Harvard College
Broad Inst Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=52105121&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=BR112015031608(A2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from PCT/US2013/074667 external-priority patent/WO2014093622A2/en
Application filed by Massachusetts Inst Technology, Harvard College, Broad Inst Inc filed Critical Massachusetts Inst Technology
Publication of BR112015031608A2 publication Critical patent/BR112015031608A2/pt

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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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Abstract

APLICAÇÃO E USO DOS SISTEMAS CRISPR-CAS, VETORES E COMPOSIÇÕES PARA DIRECIONAMENTO E TERAPIA HEPÁTICOS. A invenção provê a aplicação, manipulação e otimização de sistemas, métodos, e composições par a manipulação de sequências e/ou atividades de sequências alvo. São providos sistemas de aplicação e tecidos ou órgãos que são visados como locais de aplicação. São também providos vetores e sistemas de vetores , alguns dos quais codificam um ou mais componentes de um complexo CRISPR, bem como métodos para o desenho e uso desses vetores. Também providos são métodos de direcionamento da formação do complexo CRISPR em células eucarióticas para assegurar especificidade aumentada para reconhecimento do alvo e evitar a toxicidade e para editar ou modificar um local alvo em um local genômico de interesse para alterar ou melhorar o estado de uma doença ou uma afecção.
BR112015031608A 2013-06-17 2014-06-10 Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos BR112015031608A2 (pt)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201361836123P 2013-06-17 2013-06-17
US201361847537P 2013-07-17 2013-07-17
US201361862355P 2013-08-05 2013-08-05
US201361871301P 2013-08-28 2013-08-28
US201361915325P 2013-12-12 2013-12-12
PCT/US2013/074667 WO2014093622A2 (en) 2012-12-12 2013-12-12 Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US201461979733P 2014-04-15 2014-04-15
PCT/US2014/041804 WO2014204726A1 (en) 2013-06-17 2014-06-10 Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy

Publications (1)

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BR112015031608A2 true BR112015031608A2 (pt) 2017-08-22

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BR112015031608A BR112015031608A2 (pt) 2013-06-17 2014-06-10 Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos

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Country Link
US (3) US10577630B2 (pt)
EP (2) EP3825406A1 (pt)
JP (4) JP6738728B2 (pt)
KR (2) KR20160030187A (pt)
CN (2) CN107995927B (pt)
AU (4) AU2014281028B2 (pt)
BR (1) BR112015031608A2 (pt)
CA (1) CA2915842C (pt)
DK (1) DK3011031T3 (pt)
IL (1) IL243196B (pt)
MX (2) MX2015017312A (pt)
RU (1) RU2716420C2 (pt)
SG (2) SG10201710486XA (pt)
WO (1) WO2014204726A1 (pt)

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