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NON HUMAN ANIMALS HAVING A DISRUPTION IN A C9ORF72 LOCUS
专利权人:
发明人:
Atanasio Amanda,Ikiz Burcin,GONG GUOCHUN,LACROIX FRALISH MICHAEL L,LAI KA MAN VENUS,VALENZUELA DAVID M
申请号:
IN201717040266
公开号:
IN201717040266A
申请日:
2017.11.10
申请国别(地区):
IN
年份:
2017
代理人:
摘要:
A non-human animal model for neurodegenerative and/or inflammatory diseases is provided, which non -human an imal comprises a disruption in a C90RF72 locus. In particular, non-human animals described herein comprise a deletion of an entire coding sequence of a C90RF72 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 Gehrigs disease) and frontotemporal de mentia (FTD)), autoimmune and/or inflammatory diseases (e.g., SLE, glomerulonephritis) are also provided.
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