4.6 Article

Treatment Paradigms for Retinal and Macular Diseases Using 3-D Retina Cultures Derived From Human Reporter Pluripotent Stem Cell Lines

Journal

Publisher

ASSOC RESEARCH VISION OPHTHALMOLOGY INC
DOI: 10.1167/iovs.15-17639

Keywords

human retina development; disease modeling; photoreceptor; high throughput screening; chemical screens; drug discovery; transcriptome; next generation sequencing; three-dimensional organoid culture; fluorescent reporter

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Funding

  1. Intramural Research Program of the National Eye Institute, National Institutes of Health [ZO1 EY000450, EY000474]
  2. Japan Society for the Promotion of Science (JSPS)
  3. JSPS Postdoctoral Fellowships for Research Abroad (Kaitoku-NIH)

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We discuss the use of pluripotent stem cell lines carrying fluorescent reporters driven by retinal promoters to derive three-dimensional (3-D) retina in culture and how this system can be exploited for elucidating human retinal biology, creating disease models in a dish, and designing targeted drug screens for retinal and macular degeneration. Furthermore, we realize that stem cell investigations are labor-intensive and require extensive resources. To expedite scientific discovery by sharing of resources and to avoid duplication of efforts, we propose the formation of a Retinal Stem Cell Consortium. In the field of vision, such collaborative approaches have been enormously successful in elucidating genetic susceptibility associated with age-related macular degeneration.

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