This grant worth 150.000 € is meant for the technology transfer of findings from previous ERC actions. The Busskamp laboratory is interested in understanding the structure and function of photoreceptors in health and disease. Photoreceptors are the retinal sensory cells converting light into electrochemical signals, which are further processed by retinal interneurons, relayed to the higher brain areas and enable seeing. One of the major causes of adult visual impairment and blindness is the progressive dysfunction and death of photoreceptors due to retinal degenerative diseases. Current treatment options are still insufficient to efficiently counteract these forms of blindness. Treatment development of many drugs and therapies for retinal diseases is cumbersome: it often fails in preclinical stages because the used animal models are suboptimal for translating results from the bench to the bedside.
Therefore, within the 2015 ERC Starting Grant “ProNeurons”, the PhD student Marta Zuzic and Volker Busskamp have developed a disruptive technology to differentiate human induced pluripotent stem cells directly into photoreceptor precursor cells by overexpressing three transcription factors yielding in up to 60% in only 10 days. This technology allows fast, efficient and unlimited production of photoreceptors essential as testbeds for drug screenings, for further disease research and for photoreceptor replacement therapies. The biggest bottleneck in photoreceptor transplantation, which is currently extensively explored as a treatment option for late stages of retinal degeneration, is the lack of human photoreceptor material in sufficient quantity and quality. State-of-the-art is to dissociate so-called human-stem-cell-derived retinal organoids and isolate the photoreceptor fraction. However, these protocols are very complex and extremely long-lasting over hundreds of days in culture, which makes them very expensive.
With the help of the ERC funding, Volker Busskamp and his team plan to bring their technology closer to the market that retinal researchers and industrial partners can benefit from the human photoreceptor model system. This includes further technology and product development and refinement. "Our long-term goal is to use our technology to develop new drugs that can effectively treat late stages of retinal degeneration and blindness," Volker Busskamp emphasizes.
Contact:
Prof. Dr. Volker Busskamp
Eye Clinic at the University Hospital Bonn
Phone: +49-(0)228-28713687
Email: volker.busskamp[at]uni-bonn.de