Next step towards the prevention of blindness: CRTD researchers obtain an U.S.-patent for a method to highly efficiently produce human retinal pigment epithelium cells (RPE)

The research team around Prof. Elly Tanaka at the DFG Research Center for Regenerative Therapies Dresden (CRTD) - Cluster of Excellence at the TU Dresden succeeded in the development of a method to produce highly efficiently functional retinal pigment epithelium cells (RPE) that are crucially influencing the sense of sight. Being already under patent protection in the European Union, the method is also under protection in the U.S. since February 2016. The produced cells can be used for cell replacement therapies for hitherto incurable eye diseases as well as pharmacological screens. Dresden. Age-related macular degeneration (AMD) as well as retinitis pigmentosa (RP) represent serious visual diseases particularly for the ageing world population. Until now there are only limited therapeutic options available. These diseases lead to the death of cells that are responsible for sight (photoreceptors). One reason is a defect in the retinal pigment epithelium cells (RPE) bordering the photoreceptors (see pic. 1). This U.S. patent (US9249390 B2, 02.02.2016) as well as the EU one open up the possibility to produce highly efficiently large quantities of pure and functional human RPE tissue from pluripotent stem cells with the ultimate goal to use them for cell replacement therapies and screens to find drugs that repair the cells.

In close collaboration with the research group of Prof. Dr. Marius Ader (CRTD) the RPE cells produced by this method (see pic. 2) were already transplanted into mice and rats with deficits in their RPE. The cells integrated into the recipient's eye and took over RPE cell’s function, which protects the photoreceptors from dying. Based on these results there are first plans to extend these transplantation studies into larger animals in preparation for possible future cell replacement therapies in man. This is being done in close collaboration with Prof. Dr. Lutz E. Pillunat (http://augen.uniklinikum-dresden.de/seite.asp?ID=10) and Dr. Dirk Sandner (http://augen.uniklinikum-dresden.de/seite.asp?ID=160) of the Eye Clinic at the University Hospital Carl Gustav Carus in Dresden.

In parallel, there are efforts using these cells to identify drugs that can prevent blindness. The research groups around Prof. Elly Tanaka, Prof. Dr. Marius Ader and Dr. Mike O. Karl have already screened active substances stimulating the function of RPE cells. Further studies will follow to validate the results.

Publications:
Characterization of a mouse model with complete RPE loss and its use for RPE cell transplantation. http://www.ncbi.nlm.nih.gov/pubmed/25103259

Three-dimensional neuroepithelial culture from human embryonic stem cells and its use for quantitative conversion to retinal pigment epithelium. http://www.ncbi.nlm.nih.gov/pubmed/23358448

Founded in 2006, the DFG Research Center for Regenerative Therapies Dresden (CRTD), Cluster of Excellence at the TU Dresden has passed the third phase of the Excellence Initiative which aims at promoting top-level research and improving the quality of German universities and research institutions. The goal of the CRTD is to explore the body's self-healing potential and to develop completely new, regenerative therapies for hitherto incurable diseases. The key areas of research include haematology and immunology, diabetes, neurodegenerative diseases and bone regeneration. At the moment, eight professors and ten group leaders are working at the CRTD – integrated into an interdisciplinary network of 87 members at seven different institutions within Dresden. In addition, 21 partners from industry are supporting the network. The synergies in the network allow for a fast translation of results from basic research to clinical applications.

www.crt-dresden.de