Cholfin J A, Sobrido M J, Perlman S, Pulst S M, Geschwind D H
Department of Neurology, University of California, Los Angeles School of Medicine, USA.
Arch Neurol. 2001 Nov;58(11):1833-5. doi: 10.1001/archneur.58.11.1833.
Spinocerebellar ataxias are a group of phenotypically and genetically heterogeneous disorders characterized by progressive degeneration of the cerebellum. The expansion of a CAG repeat upstream of the PP2APR55beta gene has been recently reported as a novel cause of a dominantly inherited ataxia (SCA12) in a kindred with limb tremor as an early feature.
To explore the relative frequency of SCA12 among familial and sporadic spinocerebellar ataxias in an ethnically diverse patient population.
We used polymerase chain reaction to analyze CAG repeat size in a series of patients presenting to an ataxia clinic in California.
The SCA12 expansion was not detected in any of the cases investigated. The largest allele found had 22 repeats, a finding within the proposed nonpathogenic range. Distribution of repeat size and heterozygosity were similar to that described previously.
These results, coupled with findings in other populations, indicate that the SCA12 mutation is a rare cause of spinocerebellar degeneration. Diagnostic testing for SCA12 should be considered in patients with cerebellum disorders and an atypical clinical phenotype, especially when tremor is initially present.
脊髓小脑共济失调是一组表型和遗传异质性疾病,其特征为小脑进行性退化。最近有报道称,PP2APR55β基因上游CAG重复序列的扩增是一个以肢体震颤为早期特征的家系中显性遗传性共济失调(SCA12)的新病因。
探讨在种族多样的患者群体中,SCA12在家族性和散发性脊髓小脑共济失调中的相对发生率。
我们运用聚合酶链反应分析了一系列前往加利福尼亚州一家共济失调诊所就诊的患者的CAG重复序列长度。
在所研究的任何病例中均未检测到SCA12扩增。发现的最大等位基因有22次重复,这一结果在提议的非致病范围内。重复序列长度的分布和杂合性与先前描述的相似。
这些结果,连同其他人群中的发现,表明SCA12突变是脊髓小脑退化的罕见病因。对于患有小脑疾病且具有非典型临床表型的患者,尤其是最初出现震颤的患者,应考虑进行SCA12的诊断检测。