Human Pluripotent Stem Cell Derived Retinal Cells for Cell Therapy: Methods for the Production, Characterization, and Transplantation

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Guest Editor

Karim Ben M’Barek

Institute for Stem cell Therapy and Exploration of Monogenic diseases (I-Stem)

Karim Ben M’Barek is a research scientist at I-Stem (Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, France). He obtained his PhD in Neuroscience from Curie Institute / University Paris-Saclay (France) in 2012, where he studied the role of the protein responsible of Huntington’s disease during hippocampal neurogenesis and depressive symptoms. Then, he joined I-Stem first as a postdoc then as a research scientist to develop stem cell-based therapies for retinal degeneration. He was the first to demonstrate that the formulation of a RPE cell therapy product is crucial to achieve the best therapeutic effect in rodent. He also developed transplantation strategies in rodent as well as patented medical devices to prepare cell therapy products. The first clinical trial using human pluripotent stem cells for Retinitis Pigmentosa is based on this work. He is also expert in the differentiation of human pluripotent stem cells into retinal cells and in tissue engineering by design.

Collection Overview

Age-related Macular Degeneration and Retinitis Pigmentosa are disease conditions where retinal cells degenerate, leading ultimately to blindness. With the easy access and live imaging of the eye, cell therapy to replace degenerated cells is currently developed by a growing number of laboratories. Human pluripotent stems cells are a promising source material to produce retinal cells of interest, including photoreceptors and retinal pigment epithelium. However, once retinal cells are produced, they should be carefully characterized for a cell therapy application with a number of quality controls. In addition, several strategies have been developed to engraft the cells either as a bulk of cells or as a bioengineered tissue into the eye.

This collection aims to regroup essential methods to prepare, characterize, engraft, and detect retinal cells; with a particular focus on retinal cells derived from pluripotent stem cells.