Gold Nanoparticles in Retinoblastoma: A Promising Approach for Diagnosis and Treatment.
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BACKGROUND: Retinoblastoma (RB) is the most common intraocular malignancy in children and poses a serious threat to vision and life if not diagnosed and treated early. Traditional diagnostic methods such as fundoscopy, ultrasonography (US), and magnetic resonance imaging (MRI) offer valuable structural insights but often fail to capture early molecular changes. Similarly, conventional treatments, including chemotherapy, radiotherapy, and enucleation, are effective but limited by systemic toxicity and collateral damage to healthy ocular tissues. Recent advances in nanotechnology have introduced gold nanoparticles (AuNPs) as promising multifunctional tools in RB diagnosis and therapy. Owing to their unique optical, magnetic, and photoacoustic (PA) properties, high biocompatibility, and surface modifiability, AuNPs can be functionalized with antibodies, drugs, or nucleic acids for targeted applications. SUMMARY: This review explores the dual role of AuNPs in RB management. Diagnostically, multifunctional AuNPs paired with Fe3O4 enable precise multimodal imaging (PA imaging/US/MRI). Antibody-conjugated gold nanoclusters enhance detection of ocular biomarkers, while gold nanorods facilitate vascular imaging via photothermal optical coherence tomography. Moreover, AuNP-DNA conjugates allow ultra-sensitive genetic diagnostics, identifying single-nucleotide polymorphisms with high specificity. Therapeutically, AuNPs function as drug carriers, photothermal agents, and gene therapy vectors. They facilitate localized chemotherapy delivery, photothermal ablation of tumor cells, and gene silencing techniques targeting oncogenic pathways. KEY MESSAGES: The theranostic potential of AuNPs - integrating diagnosis and treatment - makes them ideal for personalized, minimally invasive RB care. The unique anatomical challenges of the eye, including the blood-retinal barrier, limited intraocular drug penetration, and the pediatric patient population, make AuNPs very advantageous in the setting of ocular oncology, distinguishing their theranostic potential in RB from applications in other cancers. The intraocular environment enables localized delivery, imaging-guided interventions, and reduced systemic exposure. Their ability to simultaneously improve diagnosis and enable targeted therapy may support future vision-preserving, minimally invasive treatment strategies in pediatric ocular oncology. This paper reviews the state-of-the-art applications of AuNPs in RB, highlighting their emerging role in transforming both early diagnosis and effective treatment of RB.