JOP20210133A1 - أنظمة تعديل جيني لتعديل جين منظم لموصلية التليف الكيسي عبر الغشاء (cftr) - Google Patents

أنظمة تعديل جيني لتعديل جين منظم لموصلية التليف الكيسي عبر الغشاء (cftr)

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Publication number
JOP20210133A1
JOP20210133A1 JOP/2021/0133A JOP20210133A JOP20210133A1 JO P20210133 A1 JOP20210133 A1 JO P20210133A1 JO P20210133 A JOP20210133 A JO P20210133A JO P20210133 A1 JOP20210133 A1 JO P20210133A1
Authority
JO
Jordan
Prior art keywords
gene
editing
cftr
cystic fibrosis
fibrosis transmembrane
Prior art date
Application number
JOP/2021/0133A
Other languages
English (en)
Inventor
Diego D'astolfo
Marco Weinberg
Original Assignee
Vertex Pharma
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
Application filed by Vertex Pharma filed Critical Vertex Pharma
Publication of JOP20210133A1 publication Critical patent/JOP20210133A1/ar

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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
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

يتعلق الاختراع الحالي بأنظمة تعديل جيني عالية الفعالية تشتمل على نوكلياز، RNA دليلي، و/أو قالب مانح واستخداماتها لتعديل جين منظم لموصلية التليف الكيسي عبر الغشاء (CFTR) إما في المعمل أو داخل جسم الكائن الحي.
JOP/2021/0133A 2018-12-05 2019-12-05 أنظمة تعديل جيني لتعديل جين منظم لموصلية التليف الكيسي عبر الغشاء (cftr) JOP20210133A1 (ar)

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
JOP20210133A1 true JOP20210133A1 (ar) 2023-01-30

Family

ID=69106176

Family Applications (1)

Application Number Title Priority Date Filing Date
JOP/2021/0133A JOP20210133A1 (ar) 2018-12-05 2019-12-05 أنظمة تعديل جيني لتعديل جين منظم لموصلية التليف الكيسي عبر الغشاء (cftr)

Country Status (10)

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

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
EA202191555A1 (ru) 2021-09-03
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
WO2020118073A1 (en) 2020-06-11
US20210403906A1 (en) 2021-12-30
IL283631A (en) 2021-07-29

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