Gene therapy, in which we have very high expectations to cure genetic disorders, is currently being translated from preclinical assays to the clinical practice for retinal degeneration diseases, with successful achievement up till now for the LCA, due to mutations in the RPE65 gene. This gave hope to cure RP of different genetic origins.
Currently, our gene therapy research team is focusing on gene replacement therapy, gene editing therapy and optogenetic gene therapy. With these promising therapies, though, comes relevant challenges.
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