Gene therapy of the retina is a novel tool for the treatment of inherited retinal degenerations. Preclinical studies from several groups world-wide including our own clearly show the promise of this approach to provide a cure for this potentially blinding group of diseases (Petit et al., Human Gene Ther 2016). Following a relatively fast advancement from bench to bedside, several clinical studies have been started in the immediate past and further human trials are in preparation.

While traditionally the focus in this field is on the expansion of therapeutic indications and vector development, opportunities for the optimization of treatment flow have at best received moderate attention. We believe, however, that quite substantial improvements in the efficacy and longevity of curative effects may be accomplished this way.

In this proposal, we aim at an optimization of the different steps of the treatment procedure in order to achieve a superior outcome for a given therapy. While most of these potential optimizations have at first to be evaluated preclinically, we feel that some of the presumed improvements may rather quickly find their way into clinical translation.