小基因
RNA剪接
外显子
生物
突变
遗传学
生殖系
种系突变
人类遗传学
基因
错义突变
选择性拼接
剪接体
桑格测序
表型
基因剔除小鼠
移码突变
外显子跳跃
剪接位点突变
无声突变
发病机制
作者
Liwei Sun,Yi Chen,Keya Tong,Weiwei Liu,Bei Liu,Yifan Wang,Guoning Huang,Jingyu Li
标识
DOI:10.1016/j.gendis.2025.101900
摘要
Diminished ovarian reserve (DOR) is one of the leading causes of infertility, which accounts for approximately 10% of women seeking fertility treatment. However, their genetic etiology and pathogenesis are largely unknown. Recently, cyclin N-terminal domain containing 1 ( CNTD1 ) was reported to be critical for meiosis in female mice. However, CNTD1 has not been found to be associated with reproductive diseases in humans. Here, we firstly identify the CNTD1 mutation in a DOR patient. The homozygous CNTD1 splicing mutation (NM_173478.3: c.823-2A>G) was identified in a DOR patient by whole-exome sequencing. The pathogenic effect of the identified CNTD1 splicing mutation was investigated by sequencing the transcript from the patient's primary leukocytes and minigene assay. A CRISPR/Cas9-mediated Cntd1 knockout mouse line was generated to investigate its role in ovarian function. The pathogenic mechanism of the identified CNTD1 mutation was further verified by functional studies. As a result, minigene assay and direct transcript sequencing from the patient revealed that this splicing mutation induced aberrant exon skipping. The homozygous truncating mutations in CNTD1 result in the production of a C-terminally truncated protein that cannot interact with its essential meiosis partner of proline-rich protein 19 (PRR19). Cntd1 knockout mice were characterized by dramatically reduced size of ovaries and prematurely depleted follicular pools, which indicate its role in female fertility. In conclusion, this study is the first to identify CNTD1 as a novel genetic cause for DOR patients and suggests the essential role of CNTD1 in human reproduction.
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