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《拉丁美洲一系列患者中脊髓小脑共济失调 19 型的表型谱》

The Phenotypic Spectrum of Spinocerebellar Ataxia Type 19 in a Series of Latin American Patients.

机构信息

Sección de Genética, Hospital San Juan de Dios, Santiago, Chile.

Fundación Arturo López Pérez, Santiago, Chile.

出版信息

Cerebellum. 2024 Aug;23(4):1727-1732. doi: 10.1007/s12311-023-01654-x. Epub 2024 Jan 5.

Abstract

Spinocerebellar ataxia 19 (SCA19) represents a rare autosomal dominant genetic disorder resulting in progressive ataxia and cerebellar atrophy. SCA19 is caused by variants in the KCND3 gene, which encodes a voltage-gated potassium channel subunit essential for cerebellar Purkinje cell function. We describe six cases from Chile and Mexico, representing the largest report on SCA19 in Latin America. These cases encompass a range of clinical presentations, highlighting the phenotypic variability within SCA19 from an early-onset, severe disease to a late-onset, slowly progressive condition with normal lifespan. While some patients present with pure ataxia, others also show cognitive impairment, dystonia, and other neurological symptoms. The correlations between specific KCND3 variants and phenotypic outcomes are complex and warrant further investigation. As the genomic landscape of spinocerebellar ataxias evolves, comprehensive genetic testing is becoming pivotal in improving diagnostic accuracy. This study contributes to a better understanding of the clinical spectrum of SCA19, laying the groundwork for further genotype-phenotype correlations and functional studies to elucidate the underlying pathophysiology.

摘要

脊髓小脑性共济失调 19 型(SCA19)是一种罕见的常染色体显性遗传疾病,导致进行性共济失调和小脑萎缩。SCA19 是由 KCND3 基因突变引起的,该基因编码电压门控钾通道亚基,对小脑浦肯野细胞功能至关重要。我们描述了来自智利和墨西哥的 6 个病例,这是拉丁美洲关于 SCA19 的最大报告。这些病例涵盖了一系列临床表现,突出了 SCA19 从早发性、严重疾病到晚发性、缓慢进展、寿命正常的表型变异性。虽然一些患者表现为单纯的共济失调,但其他患者也表现出认知障碍、肌张力障碍和其他神经症状。特定 KCND3 变体与表型结果之间的相关性很复杂,需要进一步研究。随着脊髓小脑性共济失调的基因组图谱不断发展,全面的基因检测对于提高诊断准确性变得至关重要。本研究有助于更好地了解 SCA19 的临床谱,为进一步的基因型-表型相关性和功能研究奠定基础,以阐明潜在的病理生理学。

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