Translational research on retinal disorders

Translationale Forschung bei Erkrankungen der Netzhaut

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Translational research on retinal disorders

Our group focuses on translational research bringing promising new gene therapies to patients with retinal disorders. Our mission is therefore to understand disease mechanisms leading to blindness, develop treatment strategies and test promising new drugs in clinical trials.

  • Understanding disease: As clinician-scientists we benefit from the unique structure of the Department of Ophthalmology with its strong links between the Institute for Ophthalmic Research and the University Eye Hospital. Using this platform, we utilize in vitro disease models, look at animal models in vivo and conduct observational trials to more precisely understand the disease process in our patients. We use this integrative approach to find new therapeutic options and define relevant outcome measures for pre-clinical and clinical trials.
  • Treatment development and optimization: We currently focus on developing and optimizing gene therapies for hereditary retinal disorders. In the lab, this entails a range of molecular biological and cellular techniques as well as work with animal models where needed (e.g. to provide proof of safety and efficacy before a therapy is used in patients). Another focus of our research addresses the challenges facing gene therapy vectors. We aim to characterize the host immune response to gene therapy, identify the leading pathways involved in host defense mechanisms and develop targeted approaches to improve safety and efficacy of gene therapeutic strategies
  • Clinical trials: We are closely involved in conducting gene therapy clinical trials in Germany. Our track record includes the world’s first clinical gene therapy trial for achromatopsia (NCT02610582) as part of the RD-CURE consortium and the first gene therapy trial for choroideremia in Germany (NCT02671539).