Nanoparticle-mediated gene therapy as a novel strategy for the treatment of retinoblastoma.

Over the last two decades, nanoparticulate delivery systems have revolutionized cancer treatment by achieving target-specific delivery, enhanced bioavailability, and improved toxicity profile. The increasing interest in nanotechnology for cancer treatment stems from the unique physicochemical properties of nanoparticles (for instance, small size, surface characteristics, etc.). Indeed, different anticancer drugs can be effectively delivered through nano-delivery systems nowadays. However, the application of such delivery systems in the arena of gene therapy remains in its infancy. Moreover, the treatment of retinoblastoma (RB), an aggressive ocular cancer of childhood, is a major problem in developing countries owing to the late diagnosis of this type of cancer. While adeno-associated virus-based delivery strategies remain the mainstay of the gene delivery method due to their high efficiency, other delivery systems, such as non-viral nanoparticles (NPs) are being developed as alternative therapeutic modalities. Indeed, different nanoparticle formulations such as lipid-based nanoparticles, polymeric nanoparticles, gold nanoparticles have displayed improved gene delivery efficiency in retinal diseases. This review article focuses on the nanoparticle mediated gene therapy approaches in the treatment of RB and highlights the attempts made to develop improved formulations for the treatment of RB. We delineate the current status of NPs as a gene delivery vehicle and cover the future perspective of this exciting field of research. Also, we discuss the achievement, challenges, and opportunities of nanomedicine to treat RB and mention novel engineering approaches that leverage our growing understanding of tumor biology and mechanisms of NPs uptake to develop more effective nanotherapeutics for RB patients.