School of Medical and Life Sciences, Chengdu University of Traditional Chinese Medicine, Chengdu, Sichuan, China.
Department of Rehabilitation, Traditional Chinese Hospital Medicine of Qing Yang District of Chengdu, Sichuan, China.
Parkinsonism Relat Disord. 2024 Sep;126:106991. doi: 10.1016/j.parkreldis.2024.106991. Epub 2024 May 3.
Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease (MJD), is the most common subtype of hereditary ataxia (HA), which is characterized by motor deficits and a lack of effective treatments, and imposes a huge physical, mental, and financial burden on patients and their families. Therefore, it is important to study the early pathogenesis of spinal cerebellar ataxia type 3 based on a mouse model for subsequent preventive treatment and seeking new therapeutic targets.
脊髓小脑共济失调 3 型（SCA3），又称 Machado-Joseph 病（MJD），是遗传性共济失调（HA）最常见的亚型，其特征为运动功能障碍，且缺乏有效的治疗方法，给患者及其家庭带来巨大的身体、心理和经济负担。因此，基于小鼠模型研究脊髓小脑共济失调 3 型的早期发病机制，对后续的预防性治疗和寻找新的治疗靶点具有重要意义。
