Simone Gana, Luisa Piccinni, Elisa Rognone, Alessia Beverino, Costanza Varesio, Alessia Asaro, Anna Pichiecchio, Enza Maria Valente
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引用次数: 0
摘要
RAS病变是一组广泛的多系统疾病,由属于RAS/MAPK通路的基因的致病性变异引起。其中,PPP1CB基因变异引起Noonan综合征样疾病伴毛发疏松2 (NSLAH2),这是一种罕见的神经、心脏、面部和骨骼受损伤的疾病,毛发疏松。我们报告一名携带复发性c146病毒的女孩C b> G (p.p pro49arg)致病变异,其表现出与以前未报道的完全共生发育相关的经典NSLH特征,可能扩大了表型。Ras蛋白在少突胶质细胞成熟和分化中的突出作用可能为我们患者观察到的髓鞘损伤提供生物学上的合理性。
Complete commissural agenesis in a child with Noonan-like syndrome with loose anagen hair 2.
RASopathies are a wide group of multisystemic disorders caused by pathogenic variants in genes belonging to the RAS/MAPK pathway. Among these, PPP1CB gene variants cause Noonan syndrome-like disorder with loose anagen hair 2 (NSLAH2), a rare condition with neuro-cardio-facio-skeletal involvement and the peculiar loose anagen hair. We report on a girl carrying the recurrent c.146 C > G (p.Pro49Arg) pathogenic variant, who presented the classical NSLH features associated with a previously unreported complete commissural agenesis, likely expanding the phenotype. The prominent role of Ras protein in oligodendrocyte maturation and differentiation might lend biological plausibility to the myelination impairment observed in our patient.
期刊介绍:
Neurogenetics publishes findings that contribute to a better understanding of the genetic basis of normal and abnormal function of the nervous system. Neurogenetic disorders are the main focus of the journal. Neurogenetics therefore includes findings in humans and other organisms that help understand neurological disease mechanisms and publishes papers from many different fields such as biophysics, cell biology, human genetics, neuroanatomy, neurochemistry, neurology, neuropathology, neurosurgery and psychiatry.
All papers submitted to Neurogenetics should be of sufficient immediate importance to justify urgent publication. They should present new scientific results. Data merely confirming previously published findings are not acceptable.