1. Fig. 1 Relevance of therapies for treating retinal degeneration and stage of disease.
Relevance of therapies for treating retinal degeneration and stage of disease. Gene replacement therapy is appropriate during the early stages of retinal degeneration when the photoreceptor cells (rods and cones) are still intact (stage I). Early intervention with gene replacement or gene editing could potentially reverse vision loss and lead to close to normal visual outcomes (stage II). Neuroprotective strategies, particularly those for preserving cones, are the best approach for treating disease where there is ongoing photoreceptor cell degeneration (stage III). Neuroprotection of cones can stave off loss of high acuity vision by protecting the fovea (stage III). Stem cell therapy, optogenetic therapy, and retinal prostheses are used to restore vision during the later stages of retinal degeneration, after the loss of cone outer segments (stage III). These approaches can be applied independently of the causal mutation and are expected to restore a low degree of vision in blind patients (stage IV). Pierre-Auguste Renoir, “Madame Henriot,” National Gallery of Art, Washington, DC.
José-Alain Sahel et al., Sci Transl Med 2019;11:eaax2324
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