Department of Surgery, Weill Cornell Medical College, New York, New York, United States of America.
PLoS Genet. 2019 May 3;15(5):e1008139. doi: 10.1371/journal.pgen.1008139. eCollection 2019 May.
Accumulating evidence indicates that paternal age correlates with disease risk in children. De novo gain-of-function mutations in the FGF-RAS-MAPK signaling pathway are known to cause a subset of genetic diseases associated with advanced paternal age, such as Apert syndrome, achondroplasia, Noonan syndrome, and Costello syndrome. It has been hypothesized that adult spermatogonial stem cells with pathogenic mutations are clonally expanded over time and propagate the mutations to offspring. However, no model system exists to interrogate mammalian germline stem cell competition in vivo. In this study, we created a lineage tracing system, which enabled undifferentiated spermatogonia with endogenous expression of HrasG12V, a known pathogenic gain-of-function mutation in RAS-MAPK signaling, to compete with their wild-type counterparts in the mouse testis. Over a year of fate analysis, neither HrasG12V-positive germ cells nor sperm exhibited a significant expansion compared to wild-type neighbors. Short-term stem cell capacity as measured by transplantation analysis was also comparable between wild-type and mutant groups. Furthermore, although constitutively active HRAS was detectable in the mutant cell lines, they did not exhibit a proliferative advantage or an enhanced response to agonist-evoked pERK signaling. These in vivo and in vitro results suggest that mouse spermatogonial stem cells are functionally resistant to a heterozygous HrasG12V mutation in the endogenous locus and that mechanisms could exist to prevent such harmful mutations from being expanded and transmitted to the next generation.
越来越多的证据表明,父亲的年龄与儿童的疾病风险有关。已知 FGF-RAS-MAPK 信号通路中的新生获得性功能突变会导致与高龄父亲相关的一部分遗传疾病,如尖嘴综合征、软骨发育不全、努南综合征和科斯特洛综合征。人们假设,具有致病性突变的成年精原干细胞随着时间的推移会克隆性扩张,并将突变传播给后代。然而,目前还没有模型系统可以在体内研究哺乳动物生殖干细胞的竞争。在这项研究中,我们创建了一个谱系追踪系统,该系统使具有内源性 HrasG12V 表达的未分化精原细胞(RAS-MAPK 信号的已知致病性获得性功能突变)能够在小鼠睾丸中与野生型细胞竞争。经过一年多的命运分析,与野生型相邻细胞相比,HrasG12V 阳性生殖细胞或精子均未出现显著扩张。通过移植分析测量的短期干细胞能力在野生型和突变型组之间也相当。此外,尽管突变细胞系中可检测到组成性激活的 HRAS,但它们并未表现出增殖优势或对激动剂诱导的 pERK 信号的增强反应。这些体内和体外结果表明,小鼠精原干细胞对同源性 HrasG12V 突变具有功能抗性,并且可能存在机制可以防止此类有害突变的扩张和传递到下一代。