PDF(Pubmed)
Abstract：
The story of acute promyelocytic leukemia (APL) discovery, physiopathology, and treatment is a unique journey, transforming the most aggressive form of leukemia to the most curable. It followed an empirical route fueled by clinical breakthroughs driving major advances in biochemistry and cell biology, including the discovery of PML nuclear bodies (PML NBs) and their central role in APL physiopathology. Beyond APL, PML NBs have emerged as key players in a wide variety of biological functions, including tumor-suppression and SUMO-initiated protein degradation, underscoring their broad importance. The APL story is an example of how clinical observations led to the incremental development of the first targeted leukemia therapy. The understanding of APL pathogenesis and the basis for cure now opens new insights in the treatment of other diseases, especially other acute myeloid leukemias.
摘要：
急性早幼粒细胞白血病（APL）的发现，病理生理学,治疗是一个独特的旅程，将最具侵袭性的白血病转化为最可治愈的白血病。它遵循了一条经验路线，由临床突破推动生物化学和细胞生物学的重大进展，包括PML核体（PMLNBs）的发现及其在APL病理生理学中的核心作用。超越APL,PMLNB已经成为各种生物学功能的关键参与者，包括肿瘤抑制和SUMO引发的蛋白质降解，强调其广泛的重要性。APL的故事是临床观察如何导致第一个靶向白血病治疗的逐步发展的一个例子。对APL发病机制和治疗基础的理解现在为其他疾病的治疗开辟了新的见解，尤其是其他急性髓系白血病。
