News vom 19.05.2017
Gene and cell based therapies take centre stage in the attempt to find effective ways to treat rare diseases. Vision loss due to inherited retinal dystrophies is a severe burden for two to four million patients worldwide and innovative strategies defeating neuronal dysfunction and cell loss are urgently needed. While the current state of the art is quite advanced in the field of experimental therapies of inherited retinal dystrophies, 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 inherited retinal dystrophies require continued work at high level.
Proposals within the Priority Programme should address at least one of the following questions: