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Volker Busskamp; Human stem-cell-derived photoreceptors for cell-based therapies. Invest. Ophthalmol. Vis. Sci. 2019;60(9):3.
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Presentation Description :
The advent of human stem cell technology now enables us to propose and test novel therapeutic interventions for replacing dysfunctional or degenerated cells and tissues. This also applies to restoring vision by transplanting stem-cell-derived RPE cells and – but still preclinical – photoreceptor approaches. In my presentation, I will focus on the generation of human photoreceptors from human stem cells for cell transplantation. For clinical approaches, one would need human photoreceptors in high quality and quantity. The gold standard is to generate photoreceptors in 2D or 3D systems followed by photoreceptor purification. However, this approach requires long timescales as photoreceptor development takes months in culture with variable quality and quantity. Therefore, my lab aims to engineer photoreceptor cell fate from human induced pluripotent stem cells (iPSCs) via directed transcriptional activation in monolayer cultures. We have discovered that inducing transcription factors in iPSCs can trigger robust and rapid neurogenesis, obtaining nearly 100% postmitotic neurons in just a few days. As the cellular ground state of iPSCs differs from retinal progenitor cell types that give rise to photoreceptors, we performed an unbiased approach testing all human transcription factors to reveal the genetic recipe for robust in vitrophotoreceptor generation.
This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.
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