PROJECT OBJECTIVESRemarkable recent advances in basic neurobiology, basic molecular biology and clinical electrophysiology have dramatically improved the prospects for finding effective treatments for tapetoretinal degenerative disorders, which is the driving force behind this joint effort to enhance our understanding of tapetoretinal degenerations and in training on how to develop rational treatment strategies for them.
The proposed program aims at building up the knowledge base in an area of strategic importance, namely treatment strategies for neuroretinal degenerative disorders. In doing so, it promotes interactions between different research laboratories. The project involves core centers and associated laboratories needed to create a critical mass, to promote interactions between basic and applied research and to ensure maximum transfer of knowledge to and from basic and applied clinical sciences.
Presently, there are dozens of genes quite well known that lead to retinal degeneration. There is an ample evidence that many forms of age-related macula degenerations are influenced not only by exogenous factors but also by genetic predisposition. The exact mechanism of how this occurs a re not very well understood. The idea to strictly base therapeutical approaches on established knowledge on the pathophysiological mechanisms that lead to retinal degeneration will result in novel therapeutic approaches, either by gene therapy, neuroprotective agents, cell transplantation or surgical techniques.
The main objective is to develop therapeutic approaches by a strong interaction among disciplines and, concerning the availability of viral vectors, ribozymes, growth factors and antiapoptotic agents to enhance the understanding of neuroprotective mechanisms and novel delivery techniques. Recent progress in this fields raises strong expectations of a major breakthrough in preventing, curing, or at least alleviating the consequences of the slow hereditary retinal degenerations as well as the negative effects of the degeneration of the macula, occurring in a large number of patients at an older age.
The individual project objectives are:
2. Training in procedures for developing therapy strategies for tapetoretinal degenerative disorders.
3. Training in assessing and understanding the pathophysiological mechanisms of neuroretinal degenerations by morphological and functional methods as well as molecular techniques.
4. Training in developing, delivering and testing novel neuroprotective agents in animal models.
5. Training in designing and performing multicenter trials in human studies including aspects of safety and ethical issues.