While the current state of the art is quite advanced in the field of experimental therapies oh IRDs, with clinical trials ongoing for some disorders and functional rescue to a certain level evident, the results of the current trials and preclinical experimentations as well as the improved understanding of the pathology of many IRDs require continued work at high level.

Within this area, the SPP 2127 will characterize the immune response within the retina following viral vector exposure, explore edema formation in retinal dystrophies to address its prevention and elimination, characterize Müller cell reactions and their potential to be used to secrete therapeutic agents for retinal neural cells and analyse the importance of pathogenicity scoring for selecting the optimal patient cohort in clinical trials.

• How does the retina react to subretinal or intravetrial injections of AAV vectors or photoreceptors and/or RPE differentiated from iPSCs?
• Since subretinal injections only treat a defined area of the retina, is the remaining untreated are having an impact (potentially negative) on the treatment outcome?
• How do the major glial cells in the retina, i.e. Müller cells, react to gene or cell therapy and how can we treat them to support therapeutic changes?