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. 2024 Aug 5;12(8):1758. doi: 10.3390/biomedicines12081758

Table 6.

Overview of gene therapy methods for the treatment of uveal melanoma.

Gene Therapy Model Used Main Findings References
Cytosine deaminase (CD) gene therapy Murine models with genetically engineered CD OCM-1 cells Introduction of the CD gene makes tumors sensitive to 5-FU. [213]
B7-H3 CAR T cells with iCas9 Human UM tissue samples and cell lines Created B7-H3 CAR T cells with an inducible caspase-9 suicide gene demonstrated a durable anti-tumor response. [214]
yCD::UPRT gene therapy In vitro primary UM cells and associated fibroblasts Transduction with yCD::UPRT gene leads to production of sEVs carrying the suicide gene, showing potential for targeting UM cells.
Needs further validation in animal models.
[215]
RNA Interference (RNAi)
siRNAs and miRNAs targeting VEGF and Bcl-2 Human UM cell line MP-38 (ATCC CRL-3296) RNA molecules, such as siRNAs and miRNAs, are utilized to target and silence genes critical for cancer growth, particularly VEGF and Bcl-2 in the context of UM. [216]
HA-coated chitosan/siRNA complexes targeting HIF-1α Human UM cell line MP-38 (ATCC CRL-3296) Demonstrated excellent cellular uptake and lysosome escape, with low cytotoxicity, effectively inhibiting the invasive potential of UM by down-regulating VEGF and HIF-1α. [216]
LncRNAs as therapeutic agents (PAUPAR, NUMB) N/A They have therapeutic potential in UM but face in vivo drug delivery challenges and lack of described interactions. [217]
miR-181a Clinically defined UM samples Identified as solely downregulated miRNA among three studies, showing significant potential as a therapeutic target in UM. [218,219,220,221]
VECTOR database VECTOR (uVeal mElanoma Correlation NeTwORk) database Published to predict RNA interactions in UM, addressing the rarity of described lncRNA–microRNA interactions and aiding in the study of RNA based therapies. [222]
siRNAs and miRNAs targeting VEGF and Bcl-2 Human UM cell line MP-38 (ATCC CRL-3296) RNA molecules, such as siRNAs and miRNAs, are utilized to target and silence genes critical for cancer growth, particularly VEGF and Bcl-2 in the context of uveal melanoma. [216]