ATANASIO, Amanda,IKIZ, Burcin,GONG, Guochun,LACROIX-FRALISH, Michael L.,LAI, Ka-Man Venus,VALENZUELA, David M.
申请号:
ES16731693
公开号:
ES2784360T3
申请日:
2016.05.26
申请国别(地区):
ES
年份:
2020
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 aC9ORF72locus. In particular, non-human animals described herein comprise a deletion of an entire coding sequence of aC9ORF72locus. 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.