Peng Xiaoyi, Song Dandan, Wang Yao, Cai Aojie, Tamang Sapana, Wang Huaili, Zhuo Zhihong
Department of Pediatrics, the First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan 450052, China.
Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2025 Apr 10;42(4):411-418. doi: 10.3760/cma.j.cn511374-20240919-00499.
To analyze the clinical characteristics and genetic etiology of a child with Christianson syndrome (CS).
A 1-year-and-5-month-old boy with CS diagnosed at the First Affiliated Hospital of Zhengzhou University in April 2021 was selected as the study subject. Clinical data were retrospectively analyzed. Peripheral blood samples were obtained from the child and his parents, followed by genomic DNA extraction and whole exome sequencing (WES). Candidate variant was validated by Sanger sequencing. This study has been approved by the Medical Ethics Committee of the Hospital of Zhengzhou University (Ethics No. 2024-KY-1103-001).
The child has manifested with seizures, microcephaly, and global developmental delay. WES revealed that he has harbored a novel de novo hemizygous nonsense variant of the SLC9A6 gene, namely c.1014G>A (p.W338*). Based on the guidelines from the American College of Medical Genetics and Genomics (ACMG), the variant was rated as pathogenic.
The hemizygous c.1014G>A nonsense variant of the SLC9A6 gene probably underlay the pathogenesis in this child. Above discovery has expanded mutational spectrum of the SLC9A6 gene and enabled definite diagnosis of the child.
分析1例克里斯蒂安森综合征(CS)患儿的临床特征及遗传病因。
选取2021年4月在郑州大学第一附属医院确诊为CS的1岁5个月男童作为研究对象。对临床资料进行回顾性分析。采集患儿及其父母的外周血样本,随后进行基因组DNA提取及全外显子组测序(WES)。通过桑格测序验证候选变异。本研究已获郑州大学附属医院医学伦理委员会批准(伦理编号:2024-KY-1103-001)。
该患儿出现癫痫发作、小头畸形及全面发育迟缓。WES显示其携带SLC9A6基因一个新的新生半合子无义变异,即c.1014G>A(p.W338*)。根据美国医学遗传学与基因组学学会(ACMG)的指南,该变异被评定为致病性变异。
SLC9A6基因的半合子c.1014G>A无义变异可能是该患儿发病的原因。上述发现扩展了SLC9A6基因的突变谱,并实现了对该患儿的明确诊断。