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Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models.
Ferla R, Pugni E, Lupo M, Tiberi P, Fioretto F, Perota A, Duchi R, Lagutina I, Gesualdo C, Rossi S, Ventrella D, Elmi A, McClinton B, Toomes C, Xu T, Molday RS, Surace EM, Simonelli F, Bacci ML, Galli C, Memon MA, Shams N, Auricchio A, Trapani I. Ferla R, et al. Sci Adv. 2025 Mar 28;11(13):eadt9354. doi: 10.1126/sciadv.adt9354. Epub 2025 Mar 26. Sci Adv. 2025. PMID: 40138422 Free PMC article.
223 results