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Constitutive PDGFRb activation drives connective tissue overgrowth through STAT5-IGF1 signaling

Kwon, H. R.; Rackley, A.; Olson, L. E.

2026-08-29 genetics
10.64898/2026.08.27.747555 bioRxiv
Show abstract

Autosomal dominant gain-of-function mutations in platelet-derived growth factor receptor beta (PDGFRb) cause overgrowth of the skeleton and other connective tissue in Kosaki overgrowth syndrome. However, the target cell type and signaling pathways underlying PDGFRb-driven overgrowth are unknown. Normal postnatal growth is controlled by pituitary-secreted growth hormone (GH), which activates the STAT5 transcriptional factor to upregulate insulin-like growth factor 1 (IGF1). To investigate the role of the GH-STAT5-IGF1 pathway in PDGFRb-related overgrowth, we generated mice with a PDGFRb gain-of-function mutation in skeletal and fibroblast lineages, which resulted in STAT5 activation and gigantism. Conditional deletion of Stat5ab in connective tissue lineages rescued skeletal overgrowth and keloid-like fibrosis in the skin. Conditional deletion of GH receptor (Ghr) did not rescue overgrowth, indicating the physiological activator of STAT5 is not required for overgrowth. However, deletion of Igf1, the STAT5 target gene, and its receptor, Igf1r, in connective tissue, rescued the overgrowth phenotype. These findings demonstrate a GHR-independent STAT5-IGF1 signaling pathway in mutant connective tissue cells, which mediates PDGFRb-driven overgrowth in mice and potentially in humans with similar PDGFRB mutations.

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