School of Medicine, Jiangsu University, 301 Xuefu Road, Zhenjiang 212013, China.
Int J Mol Sci. 2024 Oct 19;25(20):11270. doi: 10.3390/ijms252011270.
Hermansky-Pudlak syndrome (HPS)-associated pulmonary fibrosis (HPS-PF) is a progressive lung disease that is a major cause of morbidity and mortality in HPS patients. Previous studies have demonstrated that the HPS proteins play an essential role in the biogenesis and function of lysosome-related organelles (LROs) in alveolar epithelial type II (AT2) cells and found that HPS-PF is associated with dysfunction of AT2 cells and abnormal immune reactions. Despite recent advances in research on HPS and the pathology of HPS-PF, the pathological mechanisms underlying HPS-PF remain poorly understood, and no effective treatment has been established. Therefore, it is necessary to refresh the progress in the pathogenesis of HPS-PF to increase our understanding of the pathogenic mechanism of HPS-PF and develop targeted therapeutic strategies. This review summarizes the recent progress in the pathogenesis of HPS-PF provides information about the current treatment strategies for HPS-PF, and hopefully increases our understanding of the pathogenesis of HPS-PF and offers thoughts for new therapeutic interventions.
Hermansky-Pudlak 综合征（HPS）相关肺纤维化（HPS-PF）是一种进行性肺部疾病，是 HPS 患者发病和死亡的主要原因。先前的研究表明，HPS 蛋白在肺泡上皮细胞 II 型（AT2）细胞中溶酶体相关细胞器（LRO）的生物发生和功能中发挥着重要作用，并发现 HPS-PF 与 AT2 细胞功能障碍和异常免疫反应有关。尽管最近在 HPS 研究和 HPS-PF 病理学方面取得了进展，但 HPS-PF 的病理机制仍知之甚少，也没有确立有效的治疗方法。因此，有必要更新 HPS-PF 的发病机制方面的进展，以增加我们对 HPS-PF 发病机制的理解并开发靶向治疗策略。本综述总结了 HPS-PF 发病机制方面的最新进展，提供了有关 HPS-PF 目前治疗策略的信息，希望增加我们对 HPS-PF 发病机制的理解并为新的治疗干预措施提供思路。
