核糖体蛋白L10(RPL10)中的一种新型突变导致X连锁智力障碍、小脑发育不全和脊椎骨骺发育不良。
A Novel Mutation in RPL10 (Ribosomal Protein L10) Causes X-Linked Intellectual Disability, Cerebellar Hypoplasia, and Spondylo-Epiphyseal Dysplasia.
作者信息
Zanni Ginevra, Kalscheuer Vera M, Friedrich Andreas, Barresi Sabina, Alfieri Paolo, Di Capua Matteo, Haas Stefan A, Piccini Giorgia, Karl Thomas, Klauck Sabine M, Bellacchio Emanuele, Emma Francesco, Cappa Marco, Bertini Enrico, Breitenbach-Koller Lore
机构信息
Unit of Molecular Medicine for Neuromuscular and Neurodegenerative Disorders, Department of Neurosciences, Bambino Gesù Children's Hospital, IRRCS, Rome, Italy.
Department of Human Molecular Genetics, Max Planck Institute for Molecular Genetics, Berlin, Germany.
出版信息
Hum Mutat. 2015 Dec;36(12):1155-8. doi: 10.1002/humu.22860. Epub 2015 Sep 14.
RPL10 encodes ribosomal protein L10 (uL16), a highly conserved multifunctional component of the large ribosomal subunit, involved in ribosome biogenesis and function. Using X-exome resequencing, we identified a novel missense mutation (c.191C>T; p.(A64V)) in the N-terminal domain of the protein, in a family with two affected cousins presenting with X-linked intellectual disability, cerebellar hypoplasia, and spondylo-epiphyseal dysplasia (SED). We assessed the impact of the mutation on the translational capacity of the cell using yeast as model system. The mutation generates a functional ribosomal protein, able to complement the translational defects of a conditional lethal mutation of yeast rpl10. However, unlike previously reported mutations, this novel RPL10 missense mutation results in an increase in the actively translating ribosome population. Our results expand the mutational and clinical spectrum of RPL10 identifying a new genetic cause of SED and highlight the emerging role of ribosomal proteins in the pathogenesis of neurodevelopmental disorders.
RPL10编码核糖体蛋白L10(uL16),它是大核糖体亚基中高度保守的多功能成分,参与核糖体的生物合成和功能。通过X外显子重测序,我们在一个家族中发现了该蛋白N端结构域的一个新的错义突变(c.191C>T;p.(A64V)),该家族中有两个患病的表亲,表现为X连锁智力残疾、小脑发育不全和脊椎骨骺发育不良(SED)。我们以酵母为模型系统评估了该突变对细胞翻译能力的影响。该突变产生了一种功能性核糖体蛋白,能够弥补酵母rpl10条件致死突变的翻译缺陷。然而,与先前报道的突变不同,这个新的RPL10错义突变导致活跃翻译的核糖体群体增加。我们的结果扩展了RPL10的突变和临床谱,确定了SED的一个新的遗传原因,并突出了核糖体蛋白在神经发育障碍发病机制中的新作用。