Department of Immunology, Genetics and Pathology, Uppsala University, Uppsala, Sweden.
Department of Molecular Medicine, Amyloidosis Research and Treatment Center, IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy.
Amyloid. 2024 Sep;31(3):159-167. doi: 10.1080/13506129.2024.2387163. Epub 2024 Aug 5.
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.
转化研究是推动系统性淀粉样变的诊断和治疗的关键。本文总结了我们于 2023 年 9 月 25-26 日在雅典举行的国际淀粉样变学会转化研究研讨会上的演讲。文中回顾了该领域先驱者所取得的关键进展，特别关注对我们理解淀粉样物质是什么以及该物质如何影响功能至关重要的发现和进展。文中还介绍了有关轻链淀粉样变中心脏损伤机制的转化研究示例、生物标志物在改善我们对疾病生物学和患者管理的理解方面的作用，以及涉及细胞毒性的分子机制。基础研究的进展继续为新的治疗途径开辟了道路。
