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REGAIN is the acronym of a new ERC Starting Grant devoted to Dr. Knut Stieger from the University Eye Clinic in Giessen, Germany to develop a treatment strategy for a devastating blinding disorder affecting photoreceptor function.
In the project “REGAIN” the applicant Dr. Stieger proposes to develop a treatment strategy for a devastating blinding disorder affecting photoreceptor function within the first decade of life, X-linked Retinitis pigmentosa (XLRP). No treatment option exists to date. The proposed treatment strategy is based on the idea of inducing homology directed repair (HDR) of the mutation by promoting the exchange between the endogenous mutated chromosomal sequence and an exogenous repair DNA template at a double strand break (DSB) site in vivo. The treatment will be realized by co-delivery of endonucleases and the template DNA via adeno-associated virus (AAV) based gene transfer.
However, This strategy has never been applied in the retina in vivo and therefore, several parameters are unknown, i.e. the average frequency of HDR in photoreceptors, whether DNA repair will take place through HDR or not, and the average length of the DNA conversion tract during HDR. The project includes the following parts:
Results from this study will significantly advance the state of the art in targeted gene correction strategies in vivo and patients with XLRP and other hereditary disorders will potentially benefit from it through extrapolating the results for a broader application.
The European Research Council's (ERC) mission is to encourage the highest quality research in Europe through competitive funding and to support investigator-initiated frontier research across all fields of research, on the basis of scientific excellence. The ERC complements other funding activities in Europe such as those of the national research funding agencies, and is a flagship component of the 'Ideas Programme' of the European Union's Seventh Research Framework Programme (FP7).
Being 'investigator-driven', or 'bottom-up', in nature, the ERC approach allows researchers to identify new opportunities and directions in any field of research, rather than being led by priorities set by politicians. This approach ensures that funds are channelled into new and promising areas of research with a greater degree of flexibility.
More information on the ERC-Website
Knut Stieger, DVM PhD
Department of Ophthalmology
Faculty of Medicine
Justus-Liebig-University Giessen
Friedrichstr. 18
35392 Giessen
Germany
Tel: +49 641 985 43835
Fax: +49 641 985 43999