Exosome-Based Approaches in Regenerative Medicine and Targeted Therapy for Eye Malignancies: A Comprehensive Review

Diagnosing and treating ocular malignancies-such as uveal melanoma, retinoblastoma, intraocular lymphoma, and conjunctival tumors-can be very difficult given their rarity, complicated pathophysiology, and a high potential for complications that threaten vision or life. Traditional treatments such as chemotherapy, radiation, and surgery result in limited clinical value because of systemic toxicity, versatile drug resistance, and insufficient local control. Exosomes (EXOs)-naturally occurring nanoscale vesicles held in biocompatible structures-represent a uniquely advantageous platform for targeting and delivering miRNAs, proteins and/or gene editing molecules across ocular barriers to create corrective, sustained, and targeted diagnostics, drug delivery, and immune modulation. Mesenchymal stem cell-derived exosomes (MSC-EXOs) also possess regenerative potential in both animal and human models of retinal and ocular injury, engaging biological pathways involved in modulating inflammation and neuroprotection such as HMGB1 and PI3K/AKT pathways. While the use of EXOs presents a promising option for ocular treatment application, several factors complicate actual clinical translation, including standardization of isolation, scalable manufacture, and regulatory issues. In general, EXO-based nanomedicine may be a promising new direction for precision therapy and regenerative ophthalmology with the increasing introduction of synthetic and bioengineered EXOs introducing precursor paving new avenues for clinically scalable and biologically customizable EXO therapeutics.