Iwasaki Yorihiro, Reyes Monica, Jüppner Harald, Bastepe Murat
Endocrine Unit, Department of Medicine, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.
Tazuke Kofukai Medical Research Institute, Kitano Hospital, Osaka, Japan.
Nat Commun. 2025 Feb 5;16(1):1377. doi: 10.1038/s41467-025-56608-0.
Genomic imprinting controls parental allele-specific gene expression via epigenetic mechanisms. Abnormal imprinting at the GNAS gene causes multiple phenotypes, including pseudohypoparathyroidism type-1B (PHP1B), a disorder of multihormone resistance. Microdeletions affecting the neighboring STX16 gene ablate an imprinting control region (STX16-ICR) of GNAS and lead to PHP1B upon maternal but not paternal inheritance. Mechanisms behind this imprinted inheritance mode remain unknown. Here, we show that the STX16-ICR forms different chromatin conformations with each GNAS parental allele and enhances two GNAS promoters in human embryonic stem cells. When these cells differentiate toward proximal renal tubule cells, STX16-ICR loses its effect, accompanied by a transition to a somatic cell-specific GNAS imprinting status. The activity of STX16-ICR depends on an OCT4 motif, whose disruption impacts transcript levels differentially on each allele. Therefore, a biallelically active embryonic enhancer dictates GNAS imprinting via different chromatin conformations, underlying the allele-specific pathogenicity of STX16-ICR microdeletions.
基因组印记通过表观遗传机制控制亲本等位基因特异性基因表达。GNAS基因的异常印记会导致多种表型,包括1B型假性甲状旁腺功能减退症(PHP1B),这是一种多激素抵抗性疾病。影响邻近STX16基因的微缺失会消除GNAS的一个印记控制区域(STX16-ICR),并在母系而非父系遗传时导致PHP1B。这种印记遗传模式背后的机制仍然未知。在这里,我们表明STX16-ICR与每个GNAS亲本等位基因形成不同的染色质构象,并增强人胚胎干细胞中的两个GNAS启动子。当这些细胞向近端肾小管细胞分化时,STX16-ICR失去其作用,同时伴随着向体细胞特异性GNAS印记状态的转变。STX16-ICR的活性取决于一个OCT4基序,其破坏对每个等位基因的转录水平有不同影响。因此,一个双等位基因活性的胚胎增强子通过不同的染色质构象决定GNAS印记,这是STX16-ICR微缺失等位基因特异性致病性的基础。