David Heslin,Roxanne Ally,Chia-Jen Siao,Ka-Man Venus Lai,David M. Valenzuela,Aarti Sharma-Kanning,Daisuke Kajimura,Gustavo Droguett,David Frendewey,Alexander O. Mujica
申请号：
US15721517
公开号：
US10781453B2
申请日:
2017.09.29
申请国别(地区):
US
年份:
2020
代理人:
摘要：
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.
