从小鼠模型中洞察脊髓小脑共济失调 3 型/马加多-约瑟夫病的早期发病机制和治疗策略。

IF 3.1 3区医学 Q2 CLINICAL NEUROLOGY

Parkinsonism & related disorders Pub Date : 2024-09-01 DOI:10.1016/j.parkreldis.2024.106991

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引用次数: 0

摘要

脊髓小脑共济失调 3 型（SCA3）又称马查多-约瑟夫病（MJD），是遗传性共济失调（HA）最常见的亚型，以运动障碍和缺乏有效治疗为特征，给患者及其家庭带来巨大的身体、精神和经济负担。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，对于后续的预防治疗和寻找新的治疗靶点具有重要意义。

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Insight into the early pathogenesis and therapeutic strategies of spinocerebellar ataxia type 3/machado–joseph disease from mouse models

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.

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来源期刊

Parkinsonism & related disorders 医学-临床神经学

CiteScore

6.20

自引率

4.90%

发文量

292

审稿时长

39 days

期刊介绍： Parkinsonism & Related Disorders publishes the results of basic and clinical research contributing to the understanding, diagnosis and treatment of all neurodegenerative syndromes in which Parkinsonism, Essential Tremor or related movement disorders may be a feature. Regular features will include: Review Articles, Point of View articles, Full-length Articles, Short Communications, Case Reports and Letter to the Editor.