Mudassir Behjat Ul, Mudassir Mujaddid, Williams Jamal B, Agha Zehra
Translational Genomics Laboratory, Department of Biosciences, COMSATS University, Islamabad, Pakistan.
Rawalpindi Institute of Cardiology, Rawalpindi, Pakistan.
PLoS One. 2025 Jan 8;20(1):e0315597. doi: 10.1371/journal.pone.0315597. eCollection 2025.
Novel denovo variants of exome sequences are major cause of pathogenic neurodevelopmental disorders with a dominant genetic mechanism that emphasize their heterogeneity and complex phenotypes. White Sutton syndrome and Gabriele-de-Vries syndrome are congenital neuro-impairments with overlap of severe intellectual disability, microcephaly, convulsions, seizures, delayed development, dysmorphism of faces, retinal diseases, movement disorders and autistic traits. POGZ gene codes for pogo transposable element-derived zinc-finger protein and YY1 gene regulates transcription, chromatin, and RNA-binding proteins that have been associated with White Sutton and Gabriele-de-Vries syndromes, in recent data. We present probands of two unrelated consanguineous families with complicated, unexplained neurocognitive syndromic characteristics clinically undiagnosed. Objectives of the study were to identify altered genetics and protein characteristics underlying molecular pathological pathways in both the patients. Whole exome sequencing identifies novel, denovo missense variant NM_015100.4: c.776 C>T (p. Pro259Leu) in exons 19 of POGZ gene and non-frameshift variant NM_003403.5: c.141_143delGGA (p. Glu47del) in exon 1 of YY1 gene for White Sutton syndrome in eight years five-month-old girl and Gabriele-de-Vries syndrome in seven years eight months old boy residing in Rawalpindi and Chakwal districts of Punjab, Pakistan respectively. Protein modelling for identified variants predicts size and conformation modifications in mutated amino acid residues that lead to damaging effects in the conserved domains expressed as neurological pathophysiology. The present study widens the diversely ethnic and highly inbred gene pool of Punjab, Pakistan population for spontaneously originated deleterious mutations and contributes to the continuously expanding phenotypic canvas. Molecular genetic identification and personalized diagnosis for the patients suffering from complicated neurodevelopmental phenotypes, for better care, management of day-to-day activities and prolonged life span are the utmost hopes.
外显子序列的新型从头变异是具有显性遗传机制的致病性神经发育障碍的主要原因,这凸显了它们的异质性和复杂表型。怀特·萨顿综合征和加布里埃尔 - 德弗里斯综合征是先天性神经损伤,伴有严重智力残疾、小头畸形、惊厥、癫痫发作、发育迟缓、面部畸形、视网膜疾病、运动障碍和自闭症特征等重叠症状。近期数据显示,POGZ基因编码pogo转座元件衍生的锌指蛋白,YY1基因调节转录、染色质和RNA结合蛋白,这些都与怀特·萨顿综合征和加布里埃尔 - 德弗里斯综合征有关。我们报告了两个无亲缘关系的近亲家庭的先证者,他们具有复杂的、无法解释的神经认知综合征特征,临床上未被诊断出来。本研究的目的是确定这两名患者潜在分子病理途径中改变的遗传学和蛋白质特征。全外显子测序在一名8岁5个月大的女孩中发现了POGZ基因第19外显子的新型从头错义变异NM_015100.4: c.776 C>T (p. Pro259Leu),该女孩患有怀特·萨顿综合征;在一名7岁8个月大的男孩中发现了YY1基因第1外显子的非移码变异NM_003403.5: c.141_143delGGA (p. Glu47del),该男孩患有加布里埃尔 - 德弗里斯综合征,这两名患者分别来自巴基斯坦旁遮普省的拉瓦尔品第和查克瓦尔地区。对已鉴定变异的蛋白质建模预测了突变氨基酸残基的大小和构象变化,这些变化会对以神经病理生理学形式表达的保守结构域产生破坏作用。本研究拓宽了巴基斯坦旁遮普人群多样的种族和高度近亲繁殖的基因库,以发现自发产生的有害突变,并为不断扩大的表型范围做出了贡献。对于患有复杂神经发育表型的患者,进行分子遗传学鉴定和个性化诊断,以提供更好的护理、日常活动管理和延长寿命,是我们最大的期望。