BR112021011703A2 - Métodos para produzir uma célula modificada e para avaliar um candidato terapêutico para o tratamento de uma doença ou condição, animal não humano ou célula animal não humana, genoma animal não humano, gene c9orf72 de animal não humano, e, agente de nuclease - Google Patents

Métodos para produzir uma célula modificada e para avaliar um candidato terapêutico para o tratamento de uma doença ou condição, animal não humano ou célula animal não humana, genoma animal não humano, gene c9orf72 de animal não humano, e, agente de nuclease Download PDF

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Publication number
BR112021011703A2
BR112021011703A2 BR112021011703-2A BR112021011703A BR112021011703A2 BR 112021011703 A2 BR112021011703 A2 BR 112021011703A2 BR 112021011703 A BR112021011703 A BR 112021011703A BR 112021011703 A2 BR112021011703 A2 BR 112021011703A2
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BR
Brazil
Prior art keywords
sequence
cell
nuclease
repeat
repeat expansion
Prior art date
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BR112021011703-2A
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English (en)
Portuguese (pt)
Inventor
Daisuke Kajimura
Aarti Sharma-Kanning
Brittany Dubose
Gustavo Droguett
Chia-Jen Siao
Junko Kuno
David Frendewey
Brian Zambrowicz
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Regeneron Pharmaceuticals, 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 Regeneron Pharmaceuticals, Inc. filed Critical Regeneron Pharmaceuticals, Inc.
Publication of BR112021011703A2 publication Critical patent/BR112021011703A2/pt

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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K67/0276—Knock-out vertebrates
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2267/03—Animal model, e.g. for test or diseases
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    • C12Q2600/00—Oligonucleotides characterized by their use
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BR112021011703-2A 2018-12-20 2019-12-13 Métodos para produzir uma célula modificada e para avaliar um candidato terapêutico para o tratamento de uma doença ou condição, animal não humano ou célula animal não humana, genoma animal não humano, gene c9orf72 de animal não humano, e, agente de nuclease BR112021011703A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862782461P 2018-12-20 2018-12-20
US62/782,461 2018-12-20
US201962829995P 2019-04-05 2019-04-05
US62/829,995 2019-04-05
PCT/US2019/066317 WO2020131632A1 (en) 2018-12-20 2019-12-13 Nuclease-mediated repeat expansion

Publications (1)

Publication Number Publication Date
BR112021011703A2 true BR112021011703A2 (pt) 2021-08-31

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BR112021011703-2A BR112021011703A2 (pt) 2018-12-20 2019-12-13 Métodos para produzir uma célula modificada e para avaliar um candidato terapêutico para o tratamento de uma doença ou condição, animal não humano ou célula animal não humana, genoma animal não humano, gene c9orf72 de animal não humano, e, agente de nuclease

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US (2) US11690362B2 (https=)
EP (1) EP3732291A1 (https=)
JP (2) JP7449291B2 (https=)
KR (1) KR102925054B1 (https=)
CN (2) CN116064550A (https=)
AU (2) AU2019403015B2 (https=)
BR (1) BR112021011703A2 (https=)
CA (1) CA3120799A1 (https=)
IL (2) IL301193A (https=)
MX (1) MX2021007400A (https=)
SG (1) SG11202105189RA (https=)
WO (1) WO2020131632A1 (https=)

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