Ｃ９ＯＲＦ７２座位中にヘキサヌクレオチドリピート伸長を有する非ヒト動物

Japanese Patent JP7026678

B2

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.

Hesslin, David
Ally, Roxanne
Xiao, Chia-Jen
Rye, Ka-Man Venus
Valenzuela, David M.
Guo, Chungwan
La Croix-Cardistry, Michael
McDonald's, Lynn
Sharma, RT
Daisuke Kajimura
Drogget, Gustavo
Friendway, David

JP2019517273A

February 28, 2022

September 29, 2017

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REGENERON PHARMACEUTICALS, INC.

A01K67/027; C12N5/10; C12N15/09; C12Q1/02; C12Q1/6813; C12Q1/6851; C12Q1/686; G01N33/15; G01N33/50

JP2016105730A

WO2016089692A1
WO2015200334A1

Neuron,2011年,72(2),p.245-256
Neuron,2015年,88(5),p.892-901

Shusaku Yamamoto
Morishita Natsuki

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