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Cell-Based Therapy for Retinal Degenerative Disease

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Stem Cell Genetics for Biomedical Research

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Abstract

The availability of non-invasive high-resolution imaging technology, the immune suppressive nature of the subretinal space, and the existence of surgical techniques that permit transplantation surgery to be a safe procedure all render the eye an ideal organ in which to begin cell-based therapy in the central nervous system. A number of early stage clinical trials are underway to assess the safety and feasibility of cell-based therapy for retinal blindness. Cell-based therapy using embryonic stem cell derived differentiated cells (e.g., retinal pigment epithelium (RPE)), neural progenitor cells, photoreceptor precursors, and bone marrow-derived hematopoietic stem/progenitor cells all have demonstrated successful rescue and/or replacement in preclinical models of human retinal degenerative disease. Additional research is needed to identify the mechanisms that control synapse formation/disjunction (to improve photoreceptor transplant efficacy), to identify factors that limit RPE survival in areas of geographic atrophy (to improve RPE transplant efficacy in eyes with age-related macular degeneration), and to identify factors that regulate immune surveillance of the subretinal space (to improve long-term photoreceptor and RPE transplant survival).

Supported in part by the Joseph J. & Marguerite DiSepio Retina Research Fund, the New Jersey Lions Eye Research Foundation, the Eng Family Foundation, and the National Eye Institute (EY021542)

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  1. Institute of Ophthalmology and Visual Science, Rutgers-New Jersey Medical School, Rutgers University, Newark, NJ, USA

    Marco A. Zarbin

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  1. Duran i Reynals Hospital, Bellvitge Biomedical Research Institute (IDIBELL), Barcelona, Spain

    Raul Delgado-Morales

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Zarbin, M.A. (2018). Cell-Based Therapy for Retinal Degenerative Disease. In: Delgado-Morales, R. (eds) Stem Cell Genetics for Biomedical Research. Springer, Cham. https://doi.org/10.1007/978-3-319-90695-9_5

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