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. 2023 Sep 27;12(19):6229. doi: 10.3390/jcm12196229

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© 2023 by the authors.

Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).

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Potential pharmaceutical interventions for Stargardt disease (STGD1). Treatment options (blue “x”) are shown in a summarized representation of the visual cycle with mutated ATP-binding cassette, subfamily A, member 4 (ABCA4) gene resulting in the accumulation of lipofuscin in the retinal pigment epithelium (RPE). Treatment for STGD1 includes several strategies. Gene therapy includes vector delivery of human ABCA4 gene. Pharmaceutical therapies include visual cycle modulators (VCM), metformin, avacincaptad pegol, and docosahexaenoic acid (DHA) and eicosapentaenoic acid (EPA). VCM represented are retinol binding protein 4 (RBP4) antagonists, deuterated vitamin A, and retinal pigment epithelium-specific 65 kDa protein (RPE65) inhibitor (emixustat). Stem cell therapy includes human pluripotent stem cell-derived retinal pigment epithelium (hESC-RPE) transplantation for regenerating RPE layer. Created with BioRender.com.