Maria Melinda Tan, Jeanne Barbara Dy, Maria Jimena Salcedo-Arellano, Flora Tassone, Randi J Hagerman
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引用次数: 0
摘要
脆性 X 精神发育迟滞 1(FMR1)基因突变会导致儿童出现一系列发育障碍,并给老年人群带来神经退行性问题。FMR1 基因有两种类型的突变。FMR1 基因的完全突变(大于 200 个 CGG 重复序列)会导致脆性 X 综合症,这是导致智力障碍和自闭症的最常见遗传原因;而在携带者中发现的预突变(55 至 200 个 CGG 重复序列)则会导致一系列问题,这些问题与 FMR1 mRNA 水平升高有关,导致 mRNA 中毒,偶尔也会导致 FMRP 水平轻度缺乏。在突变前携带者中已发现两种疾病,即:脆性 X 相关原发性卵巢发育不全(FXPOI)和脆性 X 相关震颤/共济失调综合征(FXTAS)。最近,为了识别一组常见于预突变携带者并在共病研究中被广泛报道的相关疾病,我们提出了一个新的独特名称:脆性 X 相关神经精神障碍(FXAND)。本文将介绍一例女性预突变携带者的病例报告,该患者主要患有精神疾病,同时还伴有慢性疼痛和睡眠障碍,与脆性 X 相关神经精神障碍(FXAND)一致。
Fragile X- associated Neuropsychiatric Disorders: A Case Report.
Mutations in the Fragile X Mental Retardation 1 (FMR1) gene create a spectrum of developmental disorders in children in addition to neurodegenerative problems in older populations. Two types of mutations are recognized in the FMR1 gene. The full mutation (>200 CGG repeats) in the FMR1 gene leads to Fragile X Syndrome which is the most common inherited cause of intellectual disability and autism, while the premutation (55 to 200 CGG repeats) identified among carriers leads to a range of problems linked to elevated levels of the FMR1 mRNA leading to mRNA toxicity and occasionally mildly deficient FMRP levels. Two disorders among premutation carriers have been recognized namely: the Fragile X-associated Primary Ovarian Insufficiency (FXPOI) and Fragile X-associated Tremor/Ataxia Syndrome (FXTAS). Recently, in order to recognize a group of associated disorders commonly found in premutation carriers and extensively reported in co-morbidities studies, a new distinctive name was proposed: Fragile X-associated Neuropsychiatric Disorders (FXAND). This paper will present a case report of a female premutation carrier who has encountered predominantly psychiatric problems, but also chronic pain and sleep disturbances consistent with FXAND.
期刊介绍:
The neurological landscape is changing rapidly. From the technological perspective, advanced molecular approaches and imaging modalities have greatly increased our understanding of neurological disease, with enhanced prospects for effective treatments in common but very serious disorders such as stroke, epilepsy, multiple sclerosis and Parkinson’s disease. Nevertheless, at the same time, the healthcare community is increasingly challenged by the rise in neurodegenerative diseases consequent upon demographic changes in developed countries.