Article
Zebrafish as a Model to Evaluate a CRISPR/Cas9-Based Exon Excision Approach as a Future Treatment Option for EYS-Associated Retinitis Pigmentosa.
International journal of molecular sciences - 25 Aug 2021
Schellens Renske, de Vrieze Erik, Graave Pam, Broekman Sanne, Nagel-Wolfrum Kerstin, Peters Theo, Kremer Hannie, Collin Rob W J, van Wijk Erwin
Abstract excerpt
Retinitis pigmentosa (RP) is an inherited retinal disease (IRD) with an overall prevalence of 1 in 4000 individuals. Mutations in EYS (Eyes shut homolog) are among the most frequent causes of non-syndromic autosomal recessively inherited RP and act via a loss-of-function mechanism. In light of the recent successes for other IRDs, we investigated the therapeutic potential of exon skipping for EYS-associated RP....
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