Peng Jia, Yang Bo, Wang Handuo, Zhang Zhiying, Cui Fangying, Li Haiyu, Zhao Yueshu, Liu Ling
Medical Genetics and Prenatal Diagnosis Department, the Third Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan 450052, China.
Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2024 Aug 10;41(8):973-976. doi: 10.3760/cma.j.cn511374-20231016-00195.
To explore the clinical characteristics and variant of CREBBP gene in a fetus with Rubinstein-Taybi syndrome (RSTS).
A fetus with RSTS diagnosed at the Third Affiliated Hospital of Zhengzhou University in August 2022 was selected as the study subject. Clinical data, amniotic fluid sample of the fetus and peripheral blood samples of its parents were collected for whole exome sequencing (WES). Candidate variant was verified by Sanger sequencing.
Foot malformation, cerebellar vermis agenesis, brain agenesis, polysyndactyly of the big toes and other phenotypes were found by prenatal ultrasound. WES revealed that the fetus has harbored a heterozygous c.4684G>T (p.E1562*) variant in exon 28 of the CREBBP gene (NM_004380.3), which was de novo in origin. Based on the guidelines from the American College of Medical Genetics and Genomics (ACMG), the variant was predicted to be pathogenic (PVS1+PS2_Moderate+PM2_Supporting). After genetic counseling, the couple had opted to terminate the pregnancy and refused autopsy of the fetus.
The c.4684G>T (p.E1562*) variant of the CREBBP gene probably underlay the RSTS in this fetus. The newly discovered variant has enriched the mutational spectrum of the CREBBP gene and illustrated that WES is an efficient tool for the prenatal diagnosis of RSTS.
探讨1例鲁宾斯坦-泰比综合征(RSTS)胎儿的临床特征及CREBBP基因变异情况。
选取2022年8月在郑州大学第三附属医院诊断为RSTS的1例胎儿作为研究对象。收集该胎儿的临床资料、羊水样本及其父母的外周血样本进行全外显子组测序(WES)。候选变异通过Sanger测序进行验证。
产前超声检查发现足部畸形、小脑蚓部发育不全、脑发育不全、拇趾多指畸形等表型。WES显示该胎儿CREBBP基因(NM_004380.3)第28外显子存在杂合c.4684G>T(p.E1562*)变异,此变异为新发。根据美国医学遗传学与基因组学学会(ACMG)的指南,该变异被预测为致病性变异(PVS1+PS2_Moderate+PM2_Supporting)。经过遗传咨询,该夫妇选择终止妊娠并拒绝胎儿尸检。
CREBBP基因的c.4684G>T(p.E1562*)变异可能是该胎儿患RSTS的原因。新发现的变异丰富了CREBBP基因的突变谱,并表明WES是RSTS产前诊断的有效工具。