Abstract: With the development of precision medicine, targeted therapy, as a new biologic therapy, has become a hot research topic. This paper reviewed the advances in gene targeted therapy for uveal malignant melanoma (UM), retinoblastoma (RB) and primary vitreoretinal lymphoma (PVRL). With the mutation rate of GNAQ/GNA11 gene exceeding 90% in UM patients, GαQ and Gα11 protein inhibitors FR900359 or their derivatives may be a potential therapeutic option. Combination therapy with protein kinase C and ERK kinase inhibitors may improve the treatment response in patients with metastatic UM. Targeted therapy strategies for Yes associated protein may be an effective means to treat UM in the future. Nutlin-3a is a small molecular inhibitor of MDM2-p53 interaction, which kills retinal mother cell lines at high concentrations. SYK inhibitors are potential targets for RB treatment under development. Silencing CKS1B gene can inhibit the growth and invasion of RB cells. Ibrutinib may prevent or delay central nervous system involvement in CD79B-positive PVRL patients.