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Prestigious ERC grant for Knut Stieger

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:

  1. Establishing the HDR frequency in photoreceptors in vivo: it is planned to optimize the frequency by co-delivery of trophic factors for the stimulation of the cellular repair machinery.
  2. Inducing a bias of repair events towards HDR: it is planned to use nickases that only cut one DNA strand or will edit the expression profile of sensor proteins in the repair pathway in vitro and in vivo.
  3. Optimization of the DNA conversion tract length: the expression profiles of helicases and other repair proteins are edited in vitro and in vivo.
  4. Treatment of the PRGR2793delA mouse model: The optimized treatment settings are identified in order to test them for functional and morphological rescue effects.

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

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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

Contact and further information

Knut Stieger, DVM PhD

Department of Ophthalmology
Faculty of Medicine
Justus-Liebig-University Giessen

Friedrichstr. 18
35392 Giessen

Tel: +49 641 985 43835
Fax: +49 641 985 43999