Researchers have identified a subset of Holstein having a range of skeletal deformities, including vertebral anomalies, referred to as complex vertebral malformation due to mutations in the SLC35A3 gene. Here, we report the first case in humans of SLC35A3-related vertebral anomalies. Our patient had prenatally diagnosed anomalous vertebrae, including butterfly, and hemivertebrae throughout the spine, as well as cleft palate, micrognathia, patent foramen ovale, patent ductus arteriosus, posterior embryotoxon, short limbs, camptodactyly, talipes valgus, rocker bottom feet, and facial dysmorphism including proptosis, nevus flammeus, and a cupped left ear. Clinical exome sequencing revealed a novel missense homozygous mutation in SLC35A3. Follow-up biochemical analysis confirmed abnormal protein glycosylation, consistent with a defective Golgi UDP-GlcNAc transporter, validating the mutations. Congenital disorders of glycosylation, including SLC35A3-CDG, can present as a wide phenotypic spectrum, including skeletal dysplasia. Previously reported patients with SLC35A3-CDG have been described with syndromic autism, epilepsy, and arthrogryposis.

译文

研究人员已经确定了荷斯坦因的一个子集,该子集具有一系列骨骼畸形,包括椎骨异常,这是由于SLC35A3基因突变导致的复杂椎体畸形。在这里,我们报告人类中与SLC35A3相关的椎骨异常的第一例。我们的患者在产前被诊断出脊椎异常,包括整个脊柱中的蝴蝶和半椎骨,以及c裂,微棘突,卵圆孔未闭,动脉导管未闭,后胚胎毒素,短肢,弯曲状,触角外翻,摇臂底足和面部畸形,包括眼球突出,痣红斑和杯状左耳。临床外显子组测序显示SLC35A3中有一个新的错义纯合突变。后续生化分析证实了异常的蛋白质糖基化,与有缺陷的高尔基UDP-GlcNAc转运蛋白相一致,证实了突变。先天性糖基化疾病(包括SLC35A3-CDG)可以表现为广泛的表型谱,包括骨骼发育不良。先前报道的SLC35A3-CDG患者已被证明患有自闭症,癫痫和关节炎。

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