Lysosomes, since their discovery, have been primarily known for degrading cellular macromolecules. However, in recent studies, they have begun to emerge as crucial regulators of cell homeostasis. They are at the crossroads of catabolic and anabolic pathways and are intricately involved in cellular trafficking, nutrient signaling, energy metabolism, and immune regulation. Their involvement in such essential cellular functions has renewed clinical interest in targeting the lysosome as a novel way to treat disease, particularly cancer. Acute myeloid leukemia (AML) is an aggressive blood cancer with a low survival probability, particularly in older patients. The genomic landscape of AML has been extensively characterized but few targeted therapies (with the exception of differentiation therapy) can achieve a long-term cure. Therefore, there is an unmet need for less intensive and more tolerable therapeutic interventions. In this review, we will give an overview on the myriad of functions performed by lysosomes and their importance in malignant disease. Furthermore, we will discuss their relevance in hematopoietic cells and different ways to potentially target them in AML.

自发现溶酶体以来，溶酶体主要以降解细胞大分子而闻名。然而，在最近的研究中，它们已开始成为细胞稳态的关键调节剂。它们处于分解代谢和合成代谢途径的十字路口，并错综复杂地参与细胞运输、营养信号、能量代谢和免疫调节。它们对这些基本细胞功能的参与重新激发了临床兴趣，将靶向溶酶体作为治疗疾病（尤其是癌症）的新方法。急性髓性白血病 (AML) 是一种侵袭性血癌，存活率很低，尤其是在老年患者中。AML 的基因组景观已得到广泛表征，但很少有靶向疗法（分化疗法除外）可以实现长期治愈。所以，对低强度和更耐受的治疗干预的需求尚未得到满足。在这篇综述中，我们将概述溶酶体执行的无数功能及其在恶性疾病中的重要性。此外，我们将讨论它们在造血细胞中的相关性以及在 AML 中潜在靶向它们的不同方法。