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NON-HUMAN ANIMALS HAVING A HEXANUCLEOTIDE REPEAT EXPANSION IN A C9ORF72 LOCUS
专利权人:
REGENERON PHARMACEUTICALS, INC.
发明人:
HESLIN, David,ALLY, Roxanne,SIAO, Chia-Jen,LAI, Ka-Man, Venus,VALENZUELA, David, M.,GUO, Chunguang,LACROIX-FRALISH, Michael,MACDONALD, Lynn,SHARMA, Aarti,KAJIMURA, Daisuke,DROGUETT, Gustavo,FRENDEWEY, David
申请号:
WO2017US54551
公开号:
WO2018064600(A1)
申请日:
2017.09.29
申请国别(地区):
世界知识产权组织国际局
年份:
2018
代理人:
摘要:
A non-human animal (e.g., a rodent) model for diseases associated with a C9ORF72 heterologous hexanucleotide repeat expansion sequence is provided, which non-human animal comprises a heterologous hexanucleotide repeat (GGGGCC) in an endogenous C9ORF72 locus. A non-human animal disclosed herein comprising a heterologous hexanucleotide repeat expansion sequence comprising at least one instance, e.g., repeat, of a hexanucleotide (GGGGCC) sequence may further exhibit a characteristic and/or phenotype associated with one or more neurodegenerative disorders (e.g., amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD), etc.). Methods of identifying therapeutic candidates that may be used to prevent, delay or treat one or more neurodegenerative (e.g., amyotrophic lateral sclerosis (ALS, also referred to as Lou Gehrig's disease) and frontotemporal dementia (FTD)) are also provided.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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