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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 and Weber, Bernhard H. F. (2015) 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. Journal of Controlled Release 217, pp. 273-283.

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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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Item type:Article
Date:2015
Institutions:Medicine > Lehrstuhl für Humangenetik
Identification Number:
ValueType
10.1016/j.jconrel.2015.09.033DOI
26400864PubMed ID
Keywords:Dextran; Gene therapy; Hyaluronic acid; Non-viral vector; Solid lipid nanoparticles; X-linked juvenile retinoschisis
Dewey Decimal Classification:600 Technology > 610 Medical sciences Medicine
Status:Published
Refereed:Yes, this version has been refereed
Created at the University of Regensburg:Yes
Item ID:35521
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