EA202191555A1 - Системы редактирования генов для редактирования гена трансмембранного регулятора муковисцидоза (cftr) - Google Patents

Системы редактирования генов для редактирования гена трансмембранного регулятора муковисцидоза (cftr)

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Publication number
EA202191555A1
EA202191555A1 EA202191555A EA202191555A EA202191555A1 EA 202191555 A1 EA202191555 A1 EA 202191555A1 EA 202191555 A EA202191555 A EA 202191555A EA 202191555 A EA202191555 A EA 202191555A EA 202191555 A1 EA202191555 A1 EA 202191555A1
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EA
Eurasian Patent Office
Prior art keywords
gene
editing
cftr
regulator
fluiscidosis
Prior art date
Application number
EA202191555A
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English (en)
Inventor
Марко Вейнберг
Диего Д'Астольфо
Original Assignee
Вертекс Фармасьютикалз Инкорпорейтед
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Application filed by Вертекс Фармасьютикалз Инкорпорейтед filed Critical Вертекс Фармасьютикалз Инкорпорейтед
Publication of EA202191555A1 publication Critical patent/EA202191555A1/ru

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Abstract

В изобретении описаны высокоэффективные системы редактирования генов, содержащие нуклеазу, направляющую РНК и/или донорную матрицу, и их применение для редактирования гена трансмембранного регулятора муковисцидоза (CFTR) in vitro или in vivo.
EA202191555A 2018-12-05 2019-12-05 Системы редактирования генов для редактирования гена трансмембранного регулятора муковисцидоза (cftr) EA202191555A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862775637P 2018-12-05 2018-12-05
PCT/US2019/064718 WO2020118073A1 (en) 2018-12-05 2019-12-05 Gene-editing systems for editing a cystic fibrosis transmembrane regulator (cftr) gene

Publications (1)

Publication Number Publication Date
EA202191555A1 true EA202191555A1 (ru) 2021-09-03

Family

ID=69106176

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202191555A EA202191555A1 (ru) 2018-12-05 2019-12-05 Системы редактирования генов для редактирования гена трансмембранного регулятора муковисцидоза (cftr)

Country Status (10)

Country Link
US (1) US20210403906A1 (ru)
EP (1) EP3891283A1 (ru)
AU (1) AU2019391114A1 (ru)
BR (1) BR112021010753A2 (ru)
CA (1) CA3121781A1 (ru)
CO (1) CO2021007320A2 (ru)
EA (1) EA202191555A1 (ru)
IL (1) IL283631A (ru)
JO (1) JOP20210133A1 (ru)
WO (1) WO2020118073A1 (ru)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022008557A2 (en) * 2020-07-08 2022-01-13 UCB Biopharma SRL Modulation of cftr expression
EP4320245A1 (en) * 2021-04-05 2024-02-14 The Board Of Regents Of The University Of Texas System Compositions, methods and uses for treating cystic fibrosis and related disorders
WO2022234519A1 (en) * 2021-05-05 2022-11-10 Crispr Therapeutics Ag Compositions and methods for using sacas9 scaffold sequences

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F Zinc finger binding domains for cnn
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
AU2010327998B2 (en) 2009-12-10 2015-11-12 Iowa State University Research Foundation, Inc. TAL effector-mediated DNA modification
EP3556858A3 (en) * 2014-04-09 2020-01-22 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis

Also Published As

Publication number Publication date
AU2019391114A1 (en) 2021-06-24
EP3891283A1 (en) 2021-10-13
CO2021007320A2 (es) 2021-06-21
CA3121781A1 (en) 2020-06-11
BR112021010753A2 (pt) 2021-09-21
JOP20210133A1 (ar) 2023-01-30
WO2020118073A1 (en) 2020-06-11
US20210403906A1 (en) 2021-12-30
IL283631A (en) 2021-07-29

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