National Amyloidosis Centre, Centre for Amyloidosis and Acute Phase Proteins, Division of Medicine, University College London, Royal Free Hospital Campus, Rowland Hill Street, London NW3 2PF, UK.
Nat Rev Nephrol. 2013 Oct;9(10):574-86. doi: 10.1038/nrneph.2013.171. Epub 2013 Aug 27.
Amyloid is an abnormal extracellular fibrillar protein deposit in the tissues. In humans, more than 25 different proteins can adopt a fibrillar conformation in vivo that results in the pathognomonic tinctorial property of amyloid (that is, green birefringence when an affected tissue specimen is stained with Congo red dye and viewed by microscopy under cross-polarized light). Amyloid deposition is associated with disturbance of organ function and causes a wide variety of clinical syndromes that are classified according to the respective fibril protein precursor. Systemic amyloidosis, in which amyloid deposits are widespread and typically accumulate gradually, continues to be fatal and is responsible for about one in 1,500 deaths per year in the UK. Advances in our understanding of the pathogenesis of systemic amyloidosis have resulted in the identification of new therapeutic targets, and several drugs with novel mechanisms of action are currently under development. Meanwhile, an increased awareness of amyloidosis coupled with enhancements to existing diagnostic techniques and therapeutic strategies have already resulted in better outcomes for patients with the disease.
淀粉样物是一种异常的细胞外纤维状蛋白在组织中的沉积。在人类中，超过 25 种不同的蛋白质可以在体内形成纤维状构象，导致淀粉样物的特征性染色性质（即用刚果红染料染色的受影响组织标本在偏光显微镜下观察时呈现绿色双折射）。淀粉样物沉积与器官功能紊乱有关，并导致广泛的临床综合征，这些综合征根据各自的纤维蛋白前体进行分类。系统性淀粉样变性症中，淀粉样物沉积广泛且通常逐渐积聚，仍具有致命性，在英国，每年每 1500 例死亡中就有一例是由其引起的。对系统性淀粉样变性症发病机制的深入了解导致了新的治疗靶点的确定，目前正在开发几种具有新型作用机制的药物。同时，对淀粉样变性症的认识不断提高，加上对现有诊断技术和治疗策略的改进，已经为该疾病患者带来了更好的治疗效果。
