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Structural recovery of the retina in a retinoschisin-deficient mouse after gene replacement therapy by solid lipid nanoparticles

Apaolaza, P. S. ; del Pozo-Rodríguez, A. ; Solinís, M. A. ; Rodríguez, J. M. ; Friedrich, U. ; Torrecilla, J. ; Weber, Bernhard H. F. ; Rodríguez-Gascón, A.


X-linked juvenile retinoschisis (XLRS) is a retinal degenerative disorder caused by mutations in the RS1 gene encoding a protein termed retinoschisin. The disease is an excellent candidate for gene replacement therapy as the majority of mutations have been shown to lead to a complete deficiency of the secreted protein in the retinal structures. In this work, we have studied the ability of ...


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