The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Sep. 22, 2020
Filed:
Sep. 29, 2017
Regeneron Pharmaceuticals, Inc., Tarrytown, NY (US);
David Heslin, Closter, NJ (US);
Roxanne Ally, Briarwood, NY (US);
Chia-Jen Siao, New York, NY (US);
Ka-Man Venus Lai, Seattle, WA (US);
David M. Valenzuela, Yorktown Heights, NY (US);
Aarti Sharma-Kanning, New York, NY (US);
Daisuke Kajimura, New York, NY (US);
Gustavo Droguett, New City, NY (US);
David Frendewey, New York, NY (US);
Alexander O. Mujica, Elmsford, NY (US);
Regeneron Pharmaceuticals, Inc., Tarrytown, NY (US);
Abstract
A non-human animal (e.g., a rodent) model for diseases associated with a C9ORF72 heterologous hexanucleotide repeat expansion sequence is provided, which non-human animal comprises a heterologous hexanucleotide repeat (GGGGCC) in an endogenous C9ORF72 locus. A non-human animal disclosed herein comprising a heterologous hexanucleotide repeat expansion sequence comprising at least one instance, e.g., repeat, of a hexanucleotide (GGGGCC) sequence may further exhibit a characteristic and/or phenotype associated with one or more neurodegenerative disorders (e.g., amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD), etc.). 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)) are also provided.