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Solid lipid nanoparticle-based vectors intended for the treatment of X-linked juvenile retinoschisis by gene therapy: In vivo approaches in Rs1h-deficient mouse model

Apaolaza, P. S. ; Del Pozo-Rodríguez, A. ; Torrecilla, J. ; Rodríguez-Gascón, A. ; Rodríguez, J. M. ; Friedrich, Ulrike ; Weber, Bernhard H. F.



Abstract

X-linked juvenile retinoschisis (XLRS), which results from mutations in the gene RS1 that encodes the protein retinoschisin, is a retinal degenerative disease affecting between 1/5000 and 1/25,000 people worldwide. Currently, there is no cure for this disease and the treatment is based on the application of low-vision aids. The aim of the present work was the in vitro and in vivo evaluation of ...

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