靶向可溶性 TGF-β 因子

IF 8.4 1区 医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS
Clarissa Becher MSc , Marius Wits MSc , Frances S. de Man Prof , Gonzalo Sanchez-Duffhues PhD , Marie-Jose Goumans Prof
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引用次数: 0

摘要

肺动脉高压(PAH)是一种罕见的进行性疾病,其特征是肺动脉血管重塑、血管阻力增加以及随后的右心室肥大和右心衰竭。其诱因是转化生长因子(TGF)-β 信号转导紊乱,包括骨形态发生蛋白(BMP)受体 2 的功能缺失突变。新出现的治疗方法旨在抑制升高的 TGF-β 水平或增强减弱的内皮 BMP 信号传导。本综述旨在总结 TGF-β 超家族在 PAH 病理生物学中的作用,以及近期发现的 PAH 患者中与 TGF-β 相关的可溶性因子表达的改变,这些因子可作为潜在的生物标记物和药物靶点。讨论的重点是这些改变的因子如何指导治疗决策和监测治疗反应,通过整合诊断和治疗(即精准医疗)促进个性化患者护理。这种方法可根据患者独特的疾病特征为其量身定制治疗策略。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Targeting Soluble TGF-β Factors
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.
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来源期刊
JACC: Basic to Translational Science
JACC: Basic to Translational Science CARDIAC & CARDIOVASCULAR SYSTEMS-
CiteScore
14.20
自引率
1.00%
发文量
161
审稿时长
16 weeks
期刊介绍: JACC: Basic to Translational Science is an open access journal that is part of the renowned Journal of the American College of Cardiology (JACC). It focuses on advancing the field of Translational Cardiovascular Medicine and aims to accelerate the translation of new scientific discoveries into therapies that improve outcomes for patients with or at risk for Cardiovascular Disease. The journal covers thematic areas such as pre-clinical research, clinical trials, personalized medicine, novel drugs, devices, and biologics, proteomics, genomics, and metabolomics, as well as early phase clinical trial methodology.
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