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Subsequently, robust protein expression was observed in the RPE, photoreceptors, and Mller glia, expanding the potential of biosafe LNP-based nanocarriers for gene therapy of inherited blindness.[197] The innovative gene-editing technology CRISPR-Cas9 has been widely employed as the next generation of gene therapeutic tools

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In addition, PGA and Kyn/Trp may have potential as biomarkers for disease monitoring and risk stratification, although their clinical utility will require validation in independent and longitudinal cohorts

Treatment comprises local surgery, radiotherapy and polychemotherapy, which are associated with acute and chronic adverse effects that may compromise quality of life in survivors