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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. and Rodríguez-Gascón, A. (2016) Structural recovery of the retina in a retinoschisin-deficient mouse after gene replacement therapy by solid lipid nanoparticles. Biomaterials 90, pp. 40-49.

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Other URL: http://doi.org/10.1016/j.biomaterials.2016.03.004


Abstract

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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Item type:Article
Date:2016
Institutions:Medicine > Lehrstuhl für Humangenetik
Identification Number:
ValueType
10.1016/j.biomaterials.2016.03.004DOI
Keywords:Dextran; Gene therapy; Hyaluronic acid; Retinoschisis; Solid lipid nanoparticles; mOPS
Dewey Decimal Classification:600 Technology > 610 Medical sciences Medicine
Status:Published
Refereed:Yes, this version has been refereed
Created at the University of Regensburg:Partially
Item ID:35119
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