Curing eye diseases with gene therapy

As things stand, the eye is one of the few organs that can be successfully treated with gene therapies when diseased. Nevertheless, the therapeutic possibilities are far from exhausted. And so the Research Unit “OCU-GT: Addressing the Unmet Needs in Ocular Gene Therapy,” established by the German Research Foundation (DFG), is seeking to develop innovative approaches. “We intend to leverage our know-how in a focused manner to overcome current hurdles for the broad successful application of gene therapy to treat challenging retinal diseases,” explains LMU Professor Stylianos Michalakis, who heads the Gene Therapy of Ocular Diseases research group at LMU’s Department of Ophthalmology. Michalakis is spokesperson of the new DFG Research Unit, which includes researchers from the University of Zurich, the Technical University of Munich (TUM), and Helmholtz Munich in addition to LMU and University of Munich Hospital.

Going beyond treating symptoms

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The researchers are focusing on both genetic and acquired retinal diseases, including hitherto untreatable disorders and ones where treatment has been limited. Current gene therapy methods are based on recombinant adeno-associated viruses (AAV), which are used to target diseased cells,” explains Michalakis. “But these methods are not suitable for all ocular diseases, because they do not transfer larger genes and are unable to target certain diseased cells.” Currently available therapies for acquired retinal diseases, moreover, need to be repeatedly administered via intraocular injection and do not halt disease progression. To develop new gene therapies, the team utilizes novel AAV technologies, retinal organoids – that is, organlike structures created in the laboratory – relevant animal models, state-of-the-art non-invasive imaging techniques for retina and brain, and AI-based analytical methods. “With these tools, we want to develop concepts for therapies that not only tackle symptoms, but can cure retinal diseases with a one-time treatment,” says Professor Martin Biel, pharmacologist at LMU’s Department of Pharmacy and deputy spokesperson of OCU-GT. The Research Unit is comprised of nine subprojects, in seven of which researchers from LMU and University of Munich Hospital are involved.

The research association is jointly funded by the DFG and the Swiss National Science Foundation (SNSF). DFG Research Units enable scientists to devote themselves to current and pressing questions in their fields of expertise and establish innovative research directions. They are funded for a period of up to eight years. Clinical research units are additionally characterized by the close conjunction of scientific and clinical work.