Title:
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
Document Type and Number:
Japanese Patent JP7026678
Kind Code:
B2
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.
Inventors:
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
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
Application Number:
JP2019517273A
Publication Date:
February 28, 2022
Filing Date:
September 29, 2017
Export Citation:
Assignee:
REGENERON PHARMACEUTICALS, INC.
International Classes:
A01K67/027; C12N5/10; C12N15/09; C12Q1/02; C12Q1/6813; C12Q1/6851; C12Q1/686; G01N33/15; G01N33/50
Domestic Patent References:
JP2016105730A |
Foreign References:
WO2016089692A1 | ||||
WO2015200334A1 |
Other References:
Neuron,2011年,72(2),p.245-256
Neuron,2015年,88(5),p.892-901
Neuron,2015年,88(5),p.892-901
Attorney, Agent or Firm:
Shusaku Yamamoto
Morishita Natsuki
Morishita Natsuki
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