Preclinical studies demonstrate circRNA nanotherapy improving outcomes in retinal degeneration, suggesting potential for gene therapy in inherited retinal dystrophies.
Inherited retinal dystrophies (IRDs) are a genetically diverse group of progressive blinding disorders with limited curative options. Despite advances in gene therapy, most IRD subtypes remain untreatable due to genetic heterogeneity and delivery challenges. Circular RNAs (circRNAs), a class of stable non-coding RNAs, have emerged as novel modulators of retinal gene expression. Recent developments in nanocarrier technology enable targeted delivery of circRNA mimics and inhibitors to retinal tissues, offering a promising therapeutic alternative. These platforms utilize lipid nanoparticles, polymeric micelles, and exosomes to bypass the blood-retinal barrier and enhance cellular uptake. Preclinical studies demonstrate restoration of photoreceptor survival pathways and delayed retinal degeneration in murine IRD models. Additionally, circRNA nanotherapy has shown efficacy in related retinal conditions such as age-related macular degeneration and diabetic retinopathy. However, translational hurdles, including invasive delivery routes, off-target effects, and regulatory gaps, limit clinical adoption. Nanocarrier-mediated circRNA modulation represents a next-generation strategy for precision therapy in IRDs, with potential to reshape the future of retinal disease management.
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Habib et al. (2025) studied this question.
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