Kocere Agnese, Chiavacci Elena, Soneson Charlotte, Jacobson Seth T, Harrison Emma N, Méndez-Acevedo Kevin Manuel, MacGowan Jacalyn S, Wells Harrison H, Hiltabidle Max S, Raghunath Azhwar, Shavit Jordan A, Panáková Daniela, Williams Margot L K, Robinson Mark D, Mosimann Christian, Burger Alexa
Department of Pediatrics, Section of Developmental Biology, University of Colorado Anschutz Medical Campus, Aurora, CO, USA; Department of Molecular Life Sciences, University of Zürich, Zürich, Switzerland.
Department of Molecular Life Sciences, University of Zürich, Zürich, Switzerland.
Dev Biol. 2025 Sep 2;528:34-56. doi: 10.1016/j.ydbio.2025.08.021.
Thrombocytopenia-Absent Radius (TAR) syndrome is a rare congenital condition with reduced platelets, forelimb anomalies, and variable heart and kidney defects. TAR syndrome is caused by mutations in RBM8A/Y14, a component of the exon junction complex. How perturbing a general mRNA-processing factor causes the selective TAR Syndrome phenotypes remains unknown. Here, we connect zebrafish rbm8a perturbation to early hematopoietic defects via attenuated non-canonical Wnt/Planar Cell Polarity (PCP) signaling. In hypomorphic rbm8a zebrafish, we observe a reduction of cd41-positive thrombocytes. rbm8a-mutant zebrafish accumulate mRNAs with retained introns, including non-canonical Wnt/PCP pathway components resulting in convergent extension defects. We found that reduced rbm8a function interacts with perturbations in non-canonical Wnt/PCP pathway genes wnt5b, wnt11f2, fzd7a, and vangl2, impairing the architecture of the lateral plate mesoderm (LPM) that forms hematopoietic, cardiovascular, kidney, and forelimb skeleton progenitors. Both mutants for rbm8a and for the PCP gene vangl2 feature impaired expression of early hematopoietic/endothelial genes runx1 and gfi1aa. Together, our data propose aberrant LPM patterning and hematopoietic defects as consequence of attenuated non-canonical Wnt/PCP signaling upon reduced rbm8a function.
血小板减少伴桡骨缺失(TAR)综合征是一种罕见的先天性疾病,其特征为血小板减少、前肢异常以及心脏和肾脏的各种缺陷。TAR综合征由外显子连接复合体的一个组成部分RBM8A/Y14发生突变引起。然而,干扰一个普通的mRNA加工因子如何导致选择性的TAR综合征表型仍不清楚。在这里,我们通过减弱非经典Wnt/平面细胞极性(PCP)信号通路,将斑马鱼rbm8a的干扰与早期造血缺陷联系起来。在低表达rbm8a的斑马鱼中,我们观察到cd41阳性血小板减少。rbm8a突变的斑马鱼积累了带有保留内含子的mRNA,包括非经典Wnt/PCP信号通路成分,从而导致会聚延伸缺陷。我们发现,rbm8a功能的降低与非经典Wnt/PCP信号通路基因wnt5b、wnt11f2、fzd7a和vangl2的干扰相互作用,损害了侧板中胚层(LPM)的结构,而侧板中胚层形成造血、心血管、肾脏和前肢骨骼祖细胞。rbm8a和PCP基因vangl2的突变体均表现出早期造血/内皮基因runx1和gfi1aa的表达受损。总之,我们的数据表明,rbm8a功能降低时,非经典Wnt/PCP信号通路减弱会导致LPM模式异常和造血缺陷。