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Single-cell transcriptome deciphers key targets of thrombopoietin receptor agonists and immune microenvironment characteristics of immune thrombocytopenia - PubMed

4 hours ago
  • #single-cell transcriptomics
  • #thrombopoietin receptor agonists
  • #immune thrombocytopenia
  • Systematic integration of network pharmacology and single-cell RNA-seq analysis identified five key genes (CACNA1A, CSF1R, PKN1, CD9, DSTYK) as targets for TPO-RAs in ITP.
  • Molecular docking confirmed strong binding affinities between four TPO-RAs (romiplostim, eltrombopag, avatrombopag, hetrombopag) and these key genes, linking therapeutic effects to thrombopoiesis and immune regulation.
  • The bone marrow immune microenvironment in ITP shows rewired cell-cell communication, particularly enhanced T cell signaling and aberrant megakaryocyte-T cell interactions.
  • Pseudotime trajectory analysis revealed disrupted megakaryocyte maturation dynamics, contributing to impaired platelet production in ITP.
  • In silico knockdown experiments indicated that CACNA1A, CSF1R, and PKN1 dysregulation exacerbates neutrophil hyperactivity, while CD9 and DSTYK knockdown impairs mitotic regulation.