默克尔细胞多瘤病毒（MCPyV）是默克尔细胞癌（MCC）发展的重要因素，默克尔细胞癌是一种侵袭性皮肤癌，具有高复发率和低存活率。事实上，它是最致命的皮肤癌。 MCPyV 阳性 MCC 的确切传播途径尚不清楚，但有几个因素可能引发其发展。 MCC 的常规治疗效果并不理想，特别是对于转移患者，显然需要新的治疗方案。基因靶向疗法为 MCC 的治疗带来了巨大希望，包括使用 siRNA 和 CRISPR/Cas (C/Cas)，但关键的是尚未转化为临床试验。验证这种方法的事实是，几种 siRNA 产品已获得 FDA 许可，而 C/Cas 已进入临床试验，尽管是针对 MCC 以外的疾病。从临床前研究转向临床需要克服许多挑战。在这篇综述中，我们全面总结了目前对 MCC 的认识，特别关注 MCPyV 阳性 MCC 以及基因靶向治疗的现状。此外，我们还讨论了阻碍 MCC 研究的主要障碍并探讨了未来的前景。© 2024 作者。约翰·威利 (John Wiley) 发表的《医学病毒学评论》

Merkel cell polyomavirus (MCPyV) is a significant contributor to the development of Merkel cell carcinoma (MCC), an aggressive skin cancer with high recurrence and a low survival rate. In fact, it is the deadliest skin cancer. The precise routes of transmission for MCPyV-positive MCC remain unclear, but several factors may trigger its development. Conventional treatments for MCC are not highly effective, especially in patients with metastasis, with a clear need for new treatment options. Gene-targeted therapies hold great promise for the treatment of MCC, including the use of siRNA and CRISPR/Cas (C/Cas) but critically none have yet been translated into clinical trials. Validating this approach is the fact that several siRNA products are already FDA licenced, while C/Cas has entered clinical trial, albeit for conditions other than MCC. There are many challenges that must be overcome to move from preclinical research to the clinic. In this review, we provide a comprehensive summary of the current understanding of MCC, with a particular focus on MCPyV-positive MCC, and the status of gene-targeted therapies. Additionally, we discuss the major obstacles that impede MCC research and explore future prospects.© 2024 The Author(s). Reviews in Medical Virology published by John Wiley & Sons Ltd.