Focus10TH ANNIVERSARY SERIES

Depicting brighter possibilities for treating blindness

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Science Translational Medicine  29 May 2019:
Vol. 11, Issue 494, eaax2324
DOI: 10.1126/scitranslmed.aax2324

Figures

  • Fig. 1 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.

    CREDIT: A. KITTERMAN/SCIENCE TRANSLATIONAL MEDICINE; FIGURE CONCEPT AND DESIGN/D. DALKARA; PIERRE-AUGUSTE RENOIR/WIKIMEDIA COMMONS

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