Regeneron Pharmaceuticals, Inc.

Burcin Ikiz

Michael L LaCroix-Fralish

David M Valenzuela

Ka-Man Venus Lai

Guochun Gong

Amanda Atanasio

Susan D Croll

Michael L Lacroix-Fralish

Michael Lacroix-Fralish

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.

Non-human animals having a disruption in a C9ORF72 locus

An animal model for motor neuron dysfunction in disease, e.g., amyotrophic lateral sclerosis (ALS), comprising a genetically modified non-human animal that comprises a genetically modified DR6 allele and exhibits normal phenotypes at birth and for a few weeks or months after birth. However, as the non-human animal ages, it develops motor neuron dysfunction that presents as one or more ALS-like symptoms, which may progress rapidly after onset. Methods of identifying candidate agents that may be used to prevent, delay or treat ALS are also provided.

Non-human animal exhibiting diminished upper and lower motor neuron function and sensory perception

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Burcin Ikiz