CO2021007320A2 - Sistemas de edición génica para editar un gen regulador de la conductancia transmembrana de la fibrosis quística (cftr) - Google Patents

Sistemas de edición génica para editar un gen regulador de la conductancia transmembrana de la fibrosis quística (cftr)

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Publication number
CO2021007320A2
CO2021007320A2 CONC2021/0007320A CO2021007320A CO2021007320A2 CO 2021007320 A2 CO2021007320 A2 CO 2021007320A2 CO 2021007320 A CO2021007320 A CO 2021007320A CO 2021007320 A2 CO2021007320 A2 CO 2021007320A2
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Colombia
Prior art keywords
gene
editing
cftr
cystic fibrosis
fibrosis transmembrane
Prior art date
Application number
CONC2021/0007320A
Other languages
English (en)
Inventor
Marco Weinberg
Diego D'astolfo
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 CO2021007320A2 publication Critical patent/CO2021007320A2/es

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    • C12N15/1137—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 against enzymes
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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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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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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Abstract

RESUMEN En este documento se describen sistemas de edición génica altamente eficientes que comprenden una nucleasa, un ARN guía, y/o un donador molde y usos de estos para editar un gen regulador de la conductancia transmembrana de la fibrosis quística (CFTR) ya sea in vitro o in vivo.
CONC2021/0007320A 2018-12-05 2021-06-03 Sistemas de edición génica para editar un gen regulador de la conductancia transmembrana de la fibrosis quística (cftr) CO2021007320A2 (es)

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
CO2021007320A2 true CO2021007320A2 (es) 2021-06-21

Family

ID=69106176

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2021/0007320A CO2021007320A2 (es) 2018-12-05 2021-06-03 Sistemas de edición génica para editar un gen regulador de la conductancia transmembrana de la fibrosis quística (cftr)

Country Status (10)

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

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
CA3213308A1 (en) * 2021-04-05 2022-10-13 Daniel J. Siegwart 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
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
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
JP2005500061A (ja) 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Ｃｎｎについての亜鉛フィンガー結合ドメイン
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
US8586363B2 (en) 2009-12-10 2013-11-19 Regents Of The University Of Minnesota TAL effector-mediated DNA modification
WO2015157070A2 (en) * 2014-04-09 2015-10-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis

Also Published As

Publication number Publication date
US20210403906A1 (en) 2021-12-30
JOP20210133A1 (ar) 2023-01-30
AU2019391114A1 (en) 2021-06-24
IL283631A (en) 2021-07-29
CA3121781A1 (en) 2020-06-11
EP3891283A1 (en) 2021-10-13
WO2020118073A1 (en) 2020-06-11
EA202191555A1 (ru) 2021-09-03
BR112021010753A2 (pt) 2021-09-21

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