一系列拉丁美洲患者的脊髓小脑共济失调 19 型表型谱。

IF 2.7 3区 医学 Q3 NEUROSCIENCES Cerebellum Pub Date : 2024-08-01 Epub Date: 2024-01-05 DOI:10.1007/s12311-023-01654-x
Diana Avila-Jaque, Fernanda Martin, M Leonor Bustamante, Mariana Luna Álvarez, José Manuel Fernández, David José Dávila Ortiz de Montellano, Rosa Pardo, Diego Varela, Marcelo Miranda
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引用次数: 0

摘要

脊髓小脑共济失调 19(SCA19)是一种罕见的常染色体显性遗传疾病,会导致进行性共济失调和小脑萎缩。SCA19 由 KCND3 基因变异引起,该基因编码的电压门控钾通道亚基对小脑浦肯野细胞的功能至关重要。我们描述了来自智利和墨西哥的六个病例,这是拉丁美洲有关 SCA19 的最大规模报告。这些病例涵盖了一系列临床表现,突显了 SCA19 的表型变异性,从早期发病、病情严重到晚期发病、缓慢进展、寿命正常。一些患者表现为单纯共济失调,而另一些患者则表现为认知障碍、肌张力障碍和其他神经系统症状。特定 KCND3 变异与表型结果之间的相关性非常复杂,值得进一步研究。随着脊髓小脑性共济失调症基因组学的发展,全面的基因检测在提高诊断准确性方面变得至关重要。这项研究有助于更好地了解 SCA19 的临床谱系,为进一步开展基因型-表型相关性和功能研究以阐明潜在的病理生理学奠定基础。
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The Phenotypic Spectrum of Spinocerebellar Ataxia Type 19 in a Series of Latin American Patients.

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.

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来源期刊
Cerebellum
Cerebellum 医学-神经科学
CiteScore
6.40
自引率
14.30%
发文量
150
审稿时长
4-8 weeks
期刊介绍: Official publication of the Society for Research on the Cerebellum devoted to genetics of cerebellar ataxias, role of cerebellum in motor control and cognitive function, and amid an ageing population, diseases associated with cerebellar dysfunction. The Cerebellum is a central source for the latest developments in fundamental neurosciences including molecular and cellular biology; behavioural neurosciences and neurochemistry; genetics; fundamental and clinical neurophysiology; neurology and neuropathology; cognition and neuroimaging. The Cerebellum benefits neuroscientists in molecular and cellular biology; neurophysiologists; researchers in neurotransmission; neurologists; radiologists; paediatricians; neuropsychologists; students of neurology and psychiatry and others.
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