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:
Jul. 04, 2023
Filed:
Dec. 13, 2019
Regeneran Pharmaceuticals, Inc., Tarrytown, NY (US);
Daisuke Kajimura, New York, NY (US);
Aarti Sharma-Kanning, New York, NY (US);
Brittany Dubose, New York, NY (US);
Gustavo Droguett, New City, NY (US);
Chia-Jen Siao, New York, NY (US);
Junko Kuno, Holmes, NY (US);
David Frendewey, New York, NY (US);
Brian Zambrowicz, Sleepy Hollow, NY (US);
Regeneran Pharmaceuticals, Inc., Tarrytown, NY (US);
Abstract
Nuclease-mediated methods for expanding repeats already present at a genomic locus are provided. Non-human animal genomes, non-human animal cells, and non-human animals comprising a heterologous hexanucleotide repeat expansion sequence inserted at an endogenous C9orf72 locus and methods of making such non-human animal cells and non-human animals through nuclease-mediated repeat expansion are also provided. Methods of using the non-human animal cells or non-human animals to identify therapeutic candidates that may be used to prevent, delay or treat one or more neurodegenerative disorders associated with repeat expansion at the C9orf72 locus are also provided.