PE20212332A1 - Moduladores de la expresioon del gen de marco de lectura abierto 72 del cromosoma 9 y usos de los mismos - Google Patents

Moduladores de la expresioon del gen de marco de lectura abierto 72 del cromosoma 9 y usos de los mismos

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PE20212332A1
PE20212332A1 PE2021001757A PE2021001757A PE20212332A1 PE 20212332 A1 PE20212332 A1 PE 20212332A1 PE 2021001757 A PE2021001757 A PE 2021001757A PE 2021001757 A PE2021001757 A PE 2021001757A PE 20212332 A1 PE20212332 A1 PE 20212332A1
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Peru
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cell
gene
mutant allele
construct
domain
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PE2021001757A
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English (en)
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Joseph F Nabhan
Amrutha Pattamatta
Mohammad Samie
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Sangamo Therapeutics Inc
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Publication of PE20212332A1 publication Critical patent/PE20212332A1/es

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    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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    • 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

Referido a una proteina de fusion que comprende un dominio de proteina con dedo de zinc (ZFP) y un dominio represor de la transcripcion, en el que el dominio ZFP se une a una region diana en un segmento intronico entre los exones 1a y 1b en un alelo mutante de un gen C9orf72 humano, donde la region diana comprende mas de 30 repeticiones en tandem de G4C2 (SEC ID NO: 1). En donde la proteina de fusion reprime la transcripcion del ARNm que contiene repeticiones del alelo mutante y no reprime la transcripcion del ARNm de tipo natural del gen. Tambien se refiere a una construccion viral de acido nucleico, una celula huesped que comprende dicha construccion, un virus recombinante, una composicion farmaceutica que comprende la construccion y un metodo para inhibir la transcripcion de un alelo mutante de un gen C9orf72 en una celula humana, como una neurona, celula glial, celula ependimaria o celula neuroepitelial.
PE2021001757A 2019-04-23 2020-04-23 Moduladores de la expresioon del gen de marco de lectura abierto 72 del cromosoma 9 y usos de los mismos PE20212332A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962837523P 2019-04-23 2019-04-23
US202062964844P 2020-01-23 2020-01-23
PCT/US2020/029590 WO2020219726A1 (en) 2019-04-23 2020-04-23 Modulators of chromosome 9 open reading frame 72 gene expression and uses thereof

Publications (1)

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PE20212332A1 true PE20212332A1 (es) 2021-12-14

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US (1) US20200339638A1 (es)
EP (1) EP3958871A1 (es)
JP (1) JP2022529500A (es)
KR (1) KR20220003561A (es)
CN (1) CN113766921A (es)
AU (1) AU2020262281A1 (es)
BR (1) BR112021020868A2 (es)
CA (1) CA3137368A1 (es)
CO (1) CO2021015653A2 (es)
IL (1) IL287384A (es)
MX (1) MX2021012836A (es)
PE (1) PE20212332A1 (es)
SG (1) SG11202111443SA (es)
TW (1) TW202104249A (es)
WO (1) WO2020219726A1 (es)

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IL301193A (en) 2018-12-20 2023-05-01 Regeneron Pharma Nuclease-mediated repeat expansion

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AU2020262281A1 (en) 2021-11-04
JP2022529500A (ja) 2022-06-22
KR20220003561A (ko) 2022-01-10
CN113766921A (zh) 2021-12-07
MX2021012836A (es) 2021-12-10
TW202104249A (zh) 2021-02-01
EP3958871A1 (en) 2022-03-02
BR112021020868A2 (pt) 2021-12-14
US20200339638A1 (en) 2020-10-29
IL287384A (en) 2021-12-01
WO2020219726A1 (en) 2020-10-29
CA3137368A1 (en) 2020-10-29
SG11202111443SA (en) 2021-11-29
CO2021015653A2 (es) 2022-01-17

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