拆分手脚畸形-揭示独特的分子诊断从巴西队列。

IF 2.9 3区 医学 Q2 GENETICS & HEREDITY
Eduardo Da Cás, José Ricardo Magliocco Ceroni, Guilherme Lopes Yamamoto, Matheus Augusto Araújo Castro, Edgard França Bisneto, Alexander Augusto de Lima Jorge, Joao Bosco Oliveira Filho, Chong Ae Kim, Ana Cristina Vitorino Krepischi, Débora Romeo Bertola
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引用次数: 0

摘要

手足裂畸形(SHFM)是一种先天性肢体畸形,主要影响手和/或脚的中央射线,具有不同的表达性,不完全外显性和综合征形式。它具有遗传异质性,包括不同位点的点突变和结构变异。在巴西的一家三级中心对5名SHFM患者进行了临床评估:其中4名患者出现了额外的非骨骼表现,包括1名患者有足裂、手并指和外胚层表现。在所有个体中都发现了与SHFM相关的基因的结构变异和点突变。我们的研究结果强调了在这组骨骼疾病中观察到的遗传异质性,以及不完全外显性,这是遗传咨询的一项具有挑战性的任务。值得注意的是,一个携带复发性MAP3K20杂合变异的个体表现出与tp63相关疾病的表型,这与最近文献中报道的突出面部畸形相反,扩大了这种新发现的SHFM综合征形式的表型谱。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Split Hand-Foot Malformations-Unveiling Unique Molecular Diagnosis From a Brazilian Cohort.

Split hand-foot malformation (SHFM) is a congenital limb malformation affecting primarily the central rays of the hands and/or feet, with variable expressivity, incomplete penetrance and syndromic forms. It is genetically heterogeneous, including point mutations and structural variants in different loci. Five individuals with SHFM were clinically evaluated in a Tertiary Center in Brazil: four of them presented additional, nonskeletal findings, including one individual with split foot, hand syndactyly, and ectodermal findings. Structural variants and point mutations in genes associated with SHFM were identified in all individuals. Our results highlight genetic heterogeneity observed in this group of skeletal disorders, alongside incomplete penetrance, a challenging task imposed on genetic counseling. Of note, an individual harboring a recurrent heterozygous variant in MAP3K20 presented a phenotype reminiscent of TP63-related disorders, contrary to the one recently reported in the literature with prominent facial dysmorphisms, expanding the phenotypic spectrum of this newly recognized syndromic form of SHFM.

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来源期刊
Clinical Genetics
Clinical Genetics 医学-遗传学
CiteScore
6.50
自引率
0.00%
发文量
175
审稿时长
3-8 weeks
期刊介绍: Clinical Genetics links research to the clinic, translating advances in our understanding of the molecular basis of genetic disease for the practising clinical geneticist. The journal publishes high quality research papers, short reports, reviews and mini-reviews that connect medical genetics research with clinical practice. Topics of particular interest are: • Linking genetic variations to disease • Genome rearrangements and disease • Epigenetics and disease • The translation of genotype to phenotype • Genetics of complex disease • Management/intervention of genetic diseases • Novel therapies for genetic diseases • Developmental biology, as it relates to clinical genetics • Social science research on the psychological and behavioural aspects of living with or being at risk of genetic disease
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