TY - JOUR
T1 - Targeting Soluble TGF-β Factors
T2 - Advances in Precision Therapy for Pulmonary Arterial Hypertension
AU - Becher, Clarissa
AU - Wits, Marius
AU - de Man, Frances S.
AU - Sanchez-Duffhues, Gonzalo
AU - Goumans, Marie-Jose
N1 - Publisher Copyright:
© 2024 The Authors
PY - 2024/11
Y1 - 2024/11
N2 - 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.
AB - 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.
KW - BMPRII
KW - endothelial
KW - miRNAs
KW - small extracellular vesicles
KW - smooth muscle cell
UR - https://www.scopus.com/pages/publications/85198217033
U2 - 10.1016/j.jacbts.2024.04.005
DO - 10.1016/j.jacbts.2024.04.005
M3 - Review article
C2 - 39619136
SN - 2452-302X
VL - 9
SP - 1360
EP - 1374
JO - JACC. Basic to translational science
JF - JACC. Basic to translational science
IS - 11
ER -