Article
Cas9/sgRNA selective targeting of the P23H Rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9.PHP.B-based delivery.
Human molecular genetics - 1 Mar 2018
Giannelli Serena G, Luoni Mirko, Castoldi Valerio, Massimino Luca, Cabassi Tommaso, Angeloni Debora, Demontis Gian Carlo, Leocani Letizia, Andreazzoli Massimiliano, Broccoli Vania
Abstract excerpt
P23H is the most common mutation in the RHODOPSIN (RHO) gene leading to a dominant form of retinitis pigmentosa (RP), a rod photoreceptor degeneration that invariably causes vision loss. Specific disruption of the disease P23H RHO mutant while preserving the wild-type (WT) functional allele would be an invaluable therapy for this disease. However, various technologies tested in the past failed to achieve...
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