MX2021007400A - Expansion de repeticiones mediada por nucleasas. - Google Patents

Expansion de repeticiones mediada por nucleasas.

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Publication number
MX2021007400A
MX2021007400A MX2021007400A MX2021007400A MX2021007400A MX 2021007400 A MX2021007400 A MX 2021007400A MX 2021007400 A MX2021007400 A MX 2021007400A MX 2021007400 A MX2021007400 A MX 2021007400A MX 2021007400 A MX2021007400 A MX 2021007400A
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MX
Mexico
Prior art keywords
repeat expansion
nuclease
mediated
human animal
methods
Prior art date
Application number
MX2021007400A
Other languages
English (en)
Inventor
David Frendewey
Brian Zambrowicz
Gustavo Droguett
Junko Kuno
Daisuke Kajimura
Chia-Jen Siao
Aarti Sharma-Kanning
Brittany Dubose
Original Assignee
Regeneron 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.)
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Publication date
Application filed by Regeneron Pharma filed Critical Regeneron Pharma
Publication of MX2021007400A publication Critical patent/MX2021007400A/es

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Abstract

En la presente se proporcionan métodos para expandir repeticiones mediados por nucleasas ya presentes en un locus genómico. Asimismo se proporcionan genomas de animales no humanos, células de animales no humanos y animales no humanos que comprenden una secuencia de expansión de repeticiones de hexanucleótidos heteróloga insertada en un locus C9orf72 endógeno y métodos para hacer tales células de animales no humanos y animales no humanos a través de la expansión de repeticiones mediada por nucleasa. Asimismo se proporcionan métodos para utilizar las células de animales no humanos o los animales no humanos para identificar candidatos terapéuticos que puedan utilizarse para prevenir, retrasar o tratar uno o más trastornos neurodegenerativos asociados con la expansión de la repetición en el locus C9orf72.
MX2021007400A 2018-12-20 2019-12-13 Expansion de repeticiones mediada por nucleasas. MX2021007400A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862782461P 2018-12-20 2018-12-20
US201962829995P 2019-04-05 2019-04-05
PCT/US2019/066317 WO2020131632A1 (en) 2018-12-20 2019-12-13 Nuclease-mediated repeat expansion

Publications (1)

Publication Number Publication Date
MX2021007400A true MX2021007400A (es) 2021-07-15

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MX2021007400A MX2021007400A (es) 2018-12-20 2019-12-13 Expansion de repeticiones mediada por nucleasas.

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US (2) US11690362B2 (es)
EP (1) EP3732291A1 (es)
JP (2) JP7449291B2 (es)
KR (1) KR20210105914A (es)
CN (2) CN113423831B (es)
AU (2) AU2019403015B2 (es)
BR (1) BR112021011703A2 (es)
CA (1) CA3120799A1 (es)
IL (2) IL301193A (es)
MX (1) MX2021007400A (es)
SG (1) SG11202105189RA (es)
WO (1) WO2020131632A1 (es)

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MX2021007400A (es) 2018-12-20 2021-07-15 Regeneron Pharma Expansion de repeticiones mediada por nucleasas.
JP2024521907A (ja) 2021-06-04 2024-06-04 アルナイラム ファーマシューティカルズ， インコーポレイテッド ヒト９番染色体オープンリーディングフレーム７２（Ｃ９ＯＲＦ７２）ｉＲＮＡ剤組成物及びその使用方法
CN114736931A (zh) * 2022-01-18 2022-07-12 赛业（苏州）生物科技有限公司 一种利用CRISPR/Cas9***敲除小鼠Abhd12基因的方法和应用
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
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CN114958852A (zh) * 2022-06-09 2022-08-30 赛业（苏州）生物科技有限公司 一种Ifnar1基因敲除小鼠动物模型的构建方法及其应用

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