ATANASIO, Amanda,IKIZ, Burcin,GONG, Guochun,LACROIX-FRALISH, Michael L.,LAI, Ka-Man Venus,VALENZUELA, David M.
申请号：
EP20160731693
公开号：
EP3302048(A1)
申请日:
2016.05.26
申请国别(地区):
欧洲专利局
年份:
2018
代理人:
摘要：
A non-human animal model for neurodegenerative and/or inflammatory diseases is provided, which non-human animal comprises a disruption in a C9ORF72 locus. In particular, non-human animals described herein comprise a deletion of an entire coding sequence of a C9ORF72 locus. 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)), autoimmune and/or inflammatory diseases (e.g., SLE, glomerulonephritis) are also provided.
