神经母细胞瘤（NB）是一种交感神经系统的异质儿科肿瘤，是婴儿中最常见且致命的颅外实体恶性肿瘤。为了了解其起源和开发新型治疗性靶向疗法，人们投入了大量的努力。在这里，我们总结了在起源细胞鉴定和发育过程中发生的导致 NB 的遗传改变方面的最新进展。我们讨论了当前的治疗方案、当前和未来的方向，以确定新的治疗代谢靶点、分化剂以及个性化组合疗法，作为改善 NB 儿童生存和生活质量的潜在方法。© 作者 2024。由牛津大学出版社代表神经肿瘤学会出版。

Neuroblastoma (NB), an heterogenous pediatric tumor of the sympathetic nervous system, is the most common and deadly extracranial solid malignancy diagnosed in infants. Numerous efforts have been invested in understanding its origin and in development of novel curative targeted therapies. Here, we summarize the recent advances in the identification of the cell of origin and the genetic alterations occurring during development contributing to NB. We discuss current treatment regimens, present and future directions for identification of novel therapeutic metabolic targets, differentiation agents, as well as personalized combinatory therapies as potential approaches for improving survival and quality of life of children with NB.© The Author(s) 2024. Published by Oxford University Press on behalf of the Society for Neuro-Oncology.