ArticleMolecular therapy. Nucleic acids2026
Streamlined synthetic regulatory cassette for efficient and photoreceptor-enriched retinal gene expression.
Article in Molecular therapy. Nucleic acids, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Inherited retinal diseases (IRDs) are major causes of vision loss and often associated with the degeneration of retinal neurons, such as photoreceptors. Adeno-associated virus (AAV) vectors hold therapeutic potential for IRDs; however, their off-target expression has prompted the development of refined engineering strategies to enhance cell-type preference without compromising transgene expression. Here, we present G5mP, a streamlined 315-bp synthetic promoter-enhancer construct designed for robust retinal gene expression. G5mP integrates three components: G5, a G protein-coupled receptor kinase 1-derived enhancer; mP, a minimal promoter from phosphodiesterase 6B; and a 5'-untranslated region (UTR) derived from retinoschisin 1 (RS1). Compared with the clinically used 742-bp RIR cassette-comprising the RS1 promoter, interphotoreceptor retinoid-binding protein enhancer, and RS1-derived 5'-UTR-G5mP drove stronger overall retinal expression and showed enhanced activity within photoreceptor cells in retinal cell lines, human retinal organoids, and mouse retina without inducing detectable cytotoxicity
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