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一名德州男孩患班布里奇 - 罗佩斯综合征:病例报告及文献综述

Bainbridge-Ropers Syndrome in a Texan Boy: A Case Report and Review of the Literature.

作者信息

Siu Xiao Tania, Colombari Arce Giuliana, Rojas Marron Andreina, Benitez Guadalupe A, Schwanecke Rebecca

机构信息

Research, Larkin Community Hospital, Miami, USA.

Research, Universidad de Ciencias Médicas, San Jose, CRI.

出版信息

Cureus. 2022 Dec 24;14(12):e32902. doi: 10.7759/cureus.32902. eCollection 2022 Dec.

Abstract

Bainbridge-Ropers syndrome (BRPS) or additional sex combs-like 3 (ASXL3)-related disorder is a neurodevelopmental disorder caused by a de novo missense mutation in the ASXL3 gene found on chromosome 18. The number of BRPS cases recorded to date is less than 100. In this report, a six-year-old Texan boy with global developmental delay, aggressive behavior, insomnia, microcephaly, strabismus, facial dysmorphic features, vesicoureteral reflux (VUR), bilateral congenital renal dysplasia, gastroesophageal reflux disease (GERD), hypotonia, failure to thrive, dysphagia, and status post-gastrostomy tube was referred to Children's Health in Dallas for evaluation. The patient shares a chromosomal abnormality with his father that did not explain his clinical findings. Therefore, further tests were indicated and a whole-exome gene sequencing revealed a de novo pathogenic heterozygous mutation in the ASXL3 gene in chromosome 18q12.1 associated with autosomal dominant BRPS. To our knowledge, this is the first case of BRPS with bilateral congenital renal dysplasia and may be correlated to the presence of the ASXL3 gene in renal tissue. This discovery provides significant new information about this condition that might be essential for comprehending it.

摘要

班布里奇 - 罗佩斯综合征(BRPS)或额外性梳样蛋白3(ASXL3)相关疾病是一种神经发育障碍,由位于18号染色体上的ASXL3基因的新生错义突变引起。迄今为止记录的BRPS病例数少于100例。在本报告中,一名患有全面发育迟缓、攻击性行为、失眠、小头畸形、斜视、面部畸形特征、膀胱输尿管反流(VUR)、双侧先天性肾发育不全、胃食管反流病(GERD)、肌张力减退、生长发育迟缓、吞咽困难以及胃造瘘管置入术后的6岁德克萨斯州男孩被转诊至达拉斯的儿童健康中心进行评估。该患者与其父亲存在一种染色体异常,但这并不能解释他的临床症状。因此,需要进一步检查,全外显子基因测序揭示了18号染色体q12.1上ASXL3基因的新生致病性杂合突变,与常染色体显性BRPS相关。据我们所知,这是首例伴有双侧先天性肾发育不全的BRPS病例,可能与肾组织中ASXL3基因的存在有关。这一发现为该病症提供了重要的新信息,可能对理解该病症至关重要。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2a87/9869920/96e01a4a62b6/cureus-0014-00000032902-i01.jpg

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