| abstract |
A non-human animal model for a neurodegenerative and/or inflammatory disease comprising a disruption of the C9ORF72 locus is provided. Specifically, the non-human animals described herein comprise a deletion of the entire coding sequence of the C9ORF72 locus. Also provided are identifications useful for the prevention, delay, or treatment of one or more neurodegenerative diseases (such as amyotrophic lateral sclerosis (ALS, also known as Gregor's disease) and frontotemporal dementia (FTD)), autologous Methods of therapeutic candidates for immune and/or inflammatory diseases (eg SLE, glomerulonephritis). |