Abstract:
Translational research is key in advancing the diagnosis and therapy of systemic amyloidoses. This paper summarises our presentations at the ISA Workshop on Translation in Systemic Amyloidoses held in Athens on September 25-26, 2023. The critical advances made by the pioneers in the field are reviewed, with particular attention to the discoveries and developments of utmost importance to our understanding of what amyloid is and how the substance affects functions. Examples of translational research regarding the mechanisms of cardiac damage in light chain amyloidosis, the role of biomarkers in improving our understanding of the biology of the disease and patients\' management, and the molecular mechanisms involved in the cytotoxicity are described. Advances in basic research continue to open new therapeutic avenues.
摘要:
转化研究是推进系统性淀粉样变病诊断和治疗的关键。本文总结了我们在2023年9月25日至26日在雅典举行的ISA系统性淀粉样翻译研讨会上的演讲。回顾了该领域先驱者取得的重要进展,特别关注对我们理解淀粉样蛋白是什么以及该物质如何影响功能至关重要的发现和发展。关于轻链淀粉样变性心脏损伤机制的翻译研究实例,生物标志物在提高我们对疾病生物学和患者管理的理解中的作用,描述了细胞毒性的分子机制。基础研究的进展继续开辟新的治疗途径。