Résumé
The advent of induced pluripotent stem cell (iPSC) technology has revolutionised biomedical research. The self-renewal capacity and multi-lineage differentiation potential of these cells allows the generation of iPSC-derived human tissues, which represent an important tool for disease modelling and therapeutic studies. One domain that has highly benefited from iPSC-technology is the study of inherited retinal dystrophies (IRDs). IRDs are a clinically and genetically heterogeneous group of disorders characterized by visual impairment due to dysfunction or degeneration of the light-sensing photoreceptors of the neuroretina. The potential of iPSCs to recapitulate retinal development by differentiating into a stratified neuroretina containing photoreceptors has resulted in the modelling of an array of IRDs as well as therapeutic testing. I will present our unpublished data recapitulating the timeline and morphological characteristics of human iPSC differentiation into retinal organoids. I will also show examples of the differential phenotypes of retinal organoids derived from IRD patients as compared to controls. Lastly, I will show the potential of iPSC-derived retinal organoids for proof-of-concept studies of therapeutic strategies, such as genome editing.