Gene therapy for uveal melanoma

AI Summary

Uveal melanoma is driven by driver gene mutations and epigenetic alterations that promote oncogenesis and high morbidity and mortality.
Targeting driver genes and epigenetic pathways with gene therapy offers a feasible approach for treating primary and metastatic uveal melanoma.
Current strategies include RNA interference, non-coding RNA, gene editing, gene replacement and suicide gene therapies for targeted uveal melanoma treatment.

AI Summary

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.

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.

Basic summary

Clin Transl Oncol. 2026 Jul 14. doi: 10.1007/s12094-026-04499-y. Online ahead of print.

ABSTRACT

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.

PMID:42446821 | DOI:10.1007/s12094-026-04499-y

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