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<p>Uveal melanoma (UM) is one of the most prevalent types of intraocular melanomas, which has relatively high morbidity and mortality rates despite being a rare disease. Genetic modifications are one of the prime driving forces behind cellular growth, proliferation, and migration in UM, among which mutation of driver genes (e.g., GNA11, GNAQ, BAP1, EIF1AX, and SF3B1, etc.) and epigenetic modifications (e.g., DNA methylation, histone modifications, and non-coding RNAs) are the key drivers behind oncogenesis. A feasible treatment approach for UM could be gene therapy targeting these genetic mutations and implicated pathways. In this review, current therapeutic options for the treatment of primary and metastatic UM will be briefly discussed in the context of the major driver gene and epigenetic mutations in UM. Primary focus will be on the current state of RNA interference, non-coding RNA, gene editing, gene replacement, and suicide gene therapies for UM, including prospective targeting strategies.</p>

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Gene therapy for uveal melanoma

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年7月14日

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  • Anika Bushra,
  • Joseph Irudayaraj

摘要

Uveal melanoma (UM) is one of the most prevalent types of intraocular melanomas, which has relatively high morbidity and mortality rates despite being a rare disease. Genetic modifications are one of the prime driving forces behind cellular growth, proliferation, and migration in UM, among which mutation of driver genes (e.g., GNA11, GNAQ, BAP1, EIF1AX, and SF3B1, etc.) and epigenetic modifications (e.g., DNA methylation, histone modifications, and non-coding RNAs) are the key drivers behind oncogenesis. A feasible treatment approach for UM could be gene therapy targeting these genetic mutations and implicated pathways. In this review, current therapeutic options for the treatment of primary and metastatic UM will be briefly discussed in the context of the major driver gene and epigenetic mutations in UM. Primary focus will be on the current state of RNA interference, non-coding RNA, gene editing, gene replacement, and suicide gene therapies for UM, including prospective targeting strategies.