Retinal repair with induced pluripotent stem cells

  • Shomoukh Al-Shamekh
    Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Fla

    Department of Ophthalmology, King Abdulaziz University Hospital, King Saud University, Riyadh, Saudi Arabia
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  • Jeffrey L. Goldberg
    Reprint requests: Jeffrey L. Goldberg, Shiley Eye Center, 9415 Campus Point Dr. #0946, University of California, La Jolla, CA 92093
    Shiley Eye Center, University of California, San Diego, Calif

    Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Fla
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Published:November 11, 2013DOI:
      Retinal degeneration such as age-related macular degeneration and other inherited forms, such as Stargardt's disease and retinitis pigmentosa, and optic neuropathies including glaucoma and ischemic optic neuropathy are major causes of vision loss and blindness worldwide. Damage to retinal pigment epithelial cells and photoreceptors in the former, and to retinal ganglion cell axons in the optic nerve and their cell bodies in the retina in the latter diseases lead to the eventual death of these retinal cells, and in humans there is no endogenous replacement or repair. Cell replacement therapies provide 1 avenue to restore function in these diseases, particularly in the case of retinal repair, although there are considerable issues to overcome, including the differentiation and integration of the transplanted cells. What stem cell sources could be used for such therapies? One promising source is induced pluripotent stem cells (iPSCs), which could be drawn from an individual patient needing therapy, or generated and banked from select donors. We review developing research in the use of iPSCs for retinal cell replacement therapy.


      AMD (age-related macular degeneration), BEST1 (bestrophin 1), bFGF (basic fibroblast growth factor), ECM (extracellular matrix), FACS (fluorescent-activated cell sorting), FGF (fibroblast growth factor), hESC (human embryonic stem cell), hiPSC (human induced pluripotent stem cell), IGF-1 (insulin growth factor 1), iPSC (induced pluripotent stem cell), IRBP (interphotoreceptor retinol binding protein), RA (retinoic acid), RGC (retinal ganglion cell), RP (retinitis pigmentosa), RPC (retinal progenitor cell), RPE (retinal pigment epithelium), SHH (sonic hedgehog)
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