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Targeting Soluble TGF-β Factors: Advances in Precision Therapy for Pulmonary Arterial Hypertension

  • Leiden University Medical Center
  • Amsterdam UMC - University of Amsterdam
  • Amsterdam UMC
  • Instituto de Investigación Sanitaria del Principado de Asturias
  • Leiden University
  • Amsterdam University Medical Centers

Research output: Contribution to journalReview articleAcademicpeer-review

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Abstract

Pulmonary arterial hypertension (PAH) is a rare progressive disease characterized by pulmonary artery vascular remodeling, increased vascular resistance, and subsequent right ventricular hypertrophy and right heart failure. It is triggered by disrupted transforming growth factor (TGF)-β signaling, including loss-of-function mutations in the bone morphogenetic protein (BMP) receptor 2. Emerging treatments aim to inhibit elevated TGF-β levels or enhance diminished endothelial BMP signaling. This review aims to summarize the role of the TGF-β superfamily in the pathobiology of PAH and recent discoveries highlighting altered expression of TGF-β–related soluble factors in PAH patients that can serve as potential biomarkers and drug targets. The discussion focuses on how these altered factors can guide treatment decisions and monitor therapeutic responses, facilitating personalized patient care through the integration of diagnostics and therapy, that is, precision medicine. This approach tailors treatment strategies to individual patients based on their unique disease characteristics.
Original languageEnglish
Pages (from-to)1360-1374
Number of pages15
JournalJACC. Basic to translational science
Volume9
Issue number11
Early online date2024
DOIs
Publication statusPublished - Nov 2024

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

  1. SDG 3 - Good Health and Well-being
    SDG 3 Good Health and Well-being

Keywords

  • BMPRII
  • endothelial
  • miRNAs
  • small extracellular vesicles
  • smooth muscle cell

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