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Generation and selection of photoreceptor precursors from human-induced pluripotent stem cells for cell therapy

Abstract : Retinal degenerative diseases represent a major public health challenge, for which there are currently no effective treatments. Cell therapy represent a promising therapeutic approach, consisting in specifically replacing degenerated cells with new cells. In this perspective, pluripotent stem cells could be used as an unlimited source of retinal cells. The work presented here aimed at contributing to the development of a cell therapy product for the treatment of photoreceptor degenerative diseases. We have demonstrated the possibility to generate and store retinal organoids from human induced pluripotent stem cells (iPSCs) using raw media complying with Good Manufacturing Practice (GMP). We have characterized the surface antigen CD73 as a marker of photoreceptors in human retinal organoids derived from human iPSCs. We have established a protocol based on the magnetic labelling of CD73-positive cells allowing for the separation of a homogenous population of photoreceptors. We could demonstrate their safety by showing the absence of tumor development upon transplantation of these cells in an immune-compromised host. Finally, CD73-positive cells had the ability to survive and to reach a certain degree of maturation in a dystrophic retinal environment. Although the ability of donor cells to establish functional synaptic connections and mediate a significant rescue of the visual function remains to be assessed, this work provides an advancement for the use of iPSC-derived photoreceptors in clinical applications and for the study of photoreceptor diseases.
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Giuliana Gagliardi. Generation and selection of photoreceptor precursors from human-induced pluripotent stem cells for cell therapy. Neurons and Cognition [q-bio.NC]. Sorbonne Université, 2018. English. ⟨NNT : 2018SORUS082⟩. ⟨tel-02505074⟩



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