P. S. Apaolaza

TL;DR: The therapeutic plasmid which encodes the protein Retinoschisin was employed achieving a positive transfection in ARPE-19 cells, showing a promising application of this new non-viral system for the treatment of X-linked juvenile retinoschisis by gene therapy.

TL;DR: In this review, the main administration routes and the most studied delivery systems, viral and non-viral, for ocular gene therapy are presented and the primary ocular disease candidates to be treated with gene therapy have been reviewed, including the genetic basis and themost relevant preclinical and clinical studies.

TL;DR: The structural improvement of the retina of Rs1h-deficient mice with PR specific expression of the RS1 gene driven by the specific promoter mOPS is shown, after successful delivery via SLN-based non-viral vectors.

TL;DR: In vitro and in vivo evaluation of two different non-viral vectors based on solid lipid nanoparticles, protamine and two anionic polysaccharides, hyaluronic acid (HA) or dextran, for the treatment of X-linked juvenile retinoschisis shows for the first time a successful RS1 gene transfer to Rs1h-deficient animals using non-Viral nanocarriers, with promising results.

TL;DR: Vectors prepared with HA showed to be twice more effective than those prepared with DX, and differences in the intracellular trafficking may justify the higher efficacy of the HA-prepared vectors.