Wang Yaochun, Song Jingzhuo, Zheng Shuxing, Wang Shuhong
Center for Translational Medicine, The First Affiliated Hospital of Xi'an Jiaotong University, Xi'an 710061, Shaanxi Province, PR China.
Department of Oncology, The First Affiliated Hospital of Xi'an Jiaotong University, Xi'an 710061, Shaanxi Province, PR China.
Transl Oncol. 2025 Jan;51:102193. doi: 10.1016/j.tranon.2024.102193. Epub 2024 Nov 21.
Von Hippel-Lindau Syndrome (VHL) is a rare genetic disorder characterized by tumors in multiple organs, including the kidneys, pancreas, and central nervous system. This comprehensive review discusses the genetic basis and clinical manifestations of VHL, as well as recent advancements in understanding the molecular mechanisms that lead to tumor formation. The authors highlight the role of hypoxia-inducible factors and the ubiquitin-proteasome system in VHL-associated cancer development .The review also discusses the potential clinical implications of these findings, such as the development of targeted therapies for VHL-associated cancers. However, the authors note the challenges associated with developing effective treatments for this complex disease, including limited patient availability for clinical trials due to its rarity .Overall, this review provides valuable insights into our current understanding of VHL and offers important avenues for future research aimed at improving the diagnosis, treatment, and management of VHL patients. By illuminating the molecular underpinnings of VHL-associated cancers, this work may ultimately help to develop more effective treatments and improve outcomes for patients with this challenging disease.
冯·希佩尔-林道综合征(VHL)是一种罕见的遗传性疾病,其特征是多个器官出现肿瘤,包括肾脏、胰腺和中枢神经系统。这篇综述讨论了VHL的遗传基础和临床表现,以及在理解导致肿瘤形成的分子机制方面的最新进展。作者强调了缺氧诱导因子和泛素-蛋白酶体系统在VHL相关癌症发展中的作用。该综述还讨论了这些发现的潜在临床意义,例如针对VHL相关癌症的靶向治疗的开发。然而,作者指出了开发针对这种复杂疾病的有效治疗方法所面临的挑战,包括由于其罕见性导致临床试验的患者可用性有限。总体而言,这篇综述为我们目前对VHL的理解提供了有价值的见解,并为未来旨在改善VHL患者诊断、治疗和管理的研究提供了重要途径。通过阐明VHL相关癌症的分子基础,这项工作最终可能有助于开发更有效的治疗方法并改善患有这种具有挑战性疾病的患者的治疗效果。