Establishment of a humanized SCA2 mouse model carrying a CAA disruption preventing CAG repeat expansion in pathogenic genes

Abstract Background Spinocerebellar ataxia type 2 (SCA2) is a neurodegenerative disease marked by significant clinical and genetic heterogeneity, primarily caused by expanded CAG mutations in the ATXN2 gene. The unstable expansion of CAG repeats disrupts the genetic stability of animal models, which...

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Autors principals:	Yao Zhang, Yufei Li, Lin Zhang, Zhaoqing Li, Keqin Lin, Kai Huang, Zhaoqing Yang, Shaohui Ma, Hao Sun, Xiaochao Zhang
Format:	Artigo
Idioma:	Inglês
Publicat:	Wiley 2025-09-01
Col·lecció:	Animal Models and Experimental Medicine
Matèries:	ATXN2 CAA interruption genetic stability mouse model SCA2
Accés en línia:	https://doi.org/10.1002/ame2.70047
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