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CO2021015653A2 - Moduladores de expresion genetica de marco de lectura abierta 72 de cromosoma 9 y usos de los mismos - Google Patents

Moduladores de expresion genetica de marco de lectura abierta 72 de cromosoma 9 y usos de los mismos

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Publication number
CO2021015653A2
CO2021015653A2 CONC2021/0015653A CO2021015653A CO2021015653A2 CO 2021015653 A2 CO2021015653 A2 CO 2021015653A2 CO 2021015653 A CO2021015653 A CO 2021015653A CO 2021015653 A2 CO2021015653 A2 CO 2021015653A2
Authority
CO
Colombia
Prior art keywords
chromosome
gene expression
reading frame
open reading
expression modulators
Prior art date
Application number
CONC2021/0015653A
Other languages
English (en)
Inventor
Joseph F Nabhan
Amrutha Pattamatta
Mohammad Samie
Original Assignee
Sangamo Therapeutics 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
Application filed by Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of CO2021015653A2 publication Critical patent/CO2021015653A2/es

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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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    • C12N2750/14011—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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Abstract

La presente divulgación suministra composiciones y métodos para modular la transcripción de alelos de gen C9orf72 mutante en pacientes en necesidad de los mismos, incluyendo pacientes que tienen una enfermedad relacionada con C9orf72, tal como esclerosis lateral amiotrófica (ALS) o demencia frontotemporal (FTD).
CONC2021/0015653A 2019-04-23 2021-11-23 Moduladores de expresion genetica de marco de lectura abierta 72 de cromosoma 9 y usos de los mismos CO2021015653A2 (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)

Publication Number Publication Date
CO2021015653A2 true CO2021015653A2 (es) 2022-01-17

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US (3) US12139517B2 (es)
EP (1) EP3958871A1 (es)
JP (1) JP7618581B2 (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)
PH (1) PH12021552554A1 (es)
SG (1) SG11202111443SA (es)
TW (1) TWI862584B (es)
WO (1) WO2020219726A1 (es)

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SG11202105189RA (en) 2018-12-20 2021-06-29 Regeneron Pharma Nuclease-mediated repeat expansion

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