Wojcik Monica H, Clark Robin D, Elias Abdallah F, Genetti Casie A, Madden Jill A, Simpson Dana, Golkar Linda, Zalusky Miranda P G, Miller Angela L, Rodriguez Araceli, Goffena Joy, Dash Camille A, Damaraju Nikhita, Gibson Sophia B, Storz Sophie H R, Anderson Zachary B, Gustafson Jonas A, Thiffault Isabelle, Farrow Emily G, Pastinen Tomi, Lin Jasmine, Huang Jennifer T, Beggs Alan H, Agrawal Pankaj B, Miller David T, Miller Danny E
Boston Children's Hospital and Harvard Medical School, Boston, MA, USA; Manton Center for Orphan Disease Research, Boston Children's Hospital, Boston, MA, USA.
Division of Clinical Genetics, Loma Linda University Children's Hospital and Loma Linda University School of Medicine, Loma Linda, CA, USA.
HGG Adv. 2025 Jul 10;6(3):100468. doi: 10.1016/j.xhgg.2025.100468. Epub 2025 Jun 12.
Incontinentia pigmenti (IP) is caused by loss-of-function variants in IKBKG, with molecular genetic diagnosis complicated by a pseudogene. We describe seven individuals from three families with IP but negative clinical genetic testing in whom long-read sequencing identified causal variants, including one family with the common exon 4-10 deletion not identified by conventional clinical genetic testing. Concurrent methylation analysis explained disease severity in one individual who died from neurologic complications, identified a mosaic variant in an individual with an atypical presentation, and confirmed skewed X chromosome inactivation in an XXY individual.
色素失禁症(IP)由IKBKG功能丧失性变异引起,分子遗传学诊断因一个假基因而变得复杂。我们描述了来自三个家庭的七名患有IP但临床基因检测呈阴性的个体,通过长读长测序在这些个体中鉴定出了致病变异,其中一个家庭存在常规临床基因检测未发现的常见外显子4 - 10缺失。同时进行的甲基化分析解释了一名死于神经系统并发症个体的疾病严重程度,在一名临床表现不典型的个体中鉴定出一个嵌合变异,并在一名XXY个体中证实了X染色体失活偏斜。