Nanotechnology-enabled drug delivery for retinoblastoma: formulation strategies, ocular targeting and translational perspectives.
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Retinoblastoma is an aggressive intraocular tumor in children, with a major underlying pathogenesis driven by mutations in the RB1 gene. Despite advances in management, current treatments, including systemic chemotherapy, focal therapies, radiotherapy, and enucleation, are limited by systemic toxicity, ocular complications, risk of secondary malignancies, and reduced efficacy in advanced disease. Additionally, poor drug penetration across ocular barriers and persistent vitreous seeding remain major therapeutic challenges. Novel drug delivery approaches such as liposomes, nanoparticles, and hydrogels have been used to achieve more localized treatment and reduce side effects. With the development of precision medicines, gene- and RNA-based therapies targeting genetic defects are promising approaches, but their targeting remains challenging. Immune checkpoint inhibitors, as well as CAR-T cells, are being explored to restore antitumor immunity and overcome tumor immune escape. Therapeutic flexibility is enhanced by complementary natural products with anti-proliferative and epigenetic effects, while combination regimens, intravitreal chemotherapy, and biomarker-guided personalized therapy support clinical translation. Future clinical research should emphasize early diagnostic biomarkers and multimodal treatment combinations to maximize survival. This review focuses on nanotechnology-based ocular drug delivery, immunotherapeutic strategies, and emerging combination approaches in RB management, highlighting their translational potential, clinical progress, and remaining challenges for global implementation and accessibility.