Stem Cell Reviews and Reports

, Volume 7, Issue 2, pp 434–445 | Cite as

Stem Cell-Based Therapeutic Applications in Retinal Degenerative Diseases

  • Yiming Huang
  • Volker Enzmann
  • Suzanne T. Ildstad


Retinal degenerative diseases that target photoreceptors or the adjacent retinal pigment epithelium (RPE) affect millions of people worldwide. Retinal degeneration (RD) is found in many different forms of retinal diseases including retinitis pigmentosa (RP), age-related macular degeneration (AMD), diabetic retinopathy, cataracts, and glaucoma. Effective treatment for retinal degeneration has been widely investigated. Gene-replacement therapy has been shown to improve visual function in inherited retinal disease. However, this treatment was less effective with advanced disease. Stem cell-based therapy is being pursued as a potential alternative approach in the treatment of retinal degenerative diseases. In this review, we will focus on stem cell-based therapies in the pipeline and summarize progress in treatment of retinal degenerative disease.


Retinal degeneration Stem cells Regeneration 



ATP-binding cassette retina


Age-related macular degeneration


Bone marrow cells


Choroidal neovascularization


Embryonic stem cells


flt3 ligand


Granulocyte colony stimulating factor


Iris pigment epithelium


Induced pluripotent stem cells


Leber congenital amaurosis


Mesenchymal stem cells


Retinal degeneration


Retinitis pigmentosa


Retinal progenitor cells


Retinal pigment epithelium


Stem cells


Very small embryonic-like stem cells



The authors thank Haval Shirwan, Larry Bozulic, and Deborah Ramsey for review of the manuscript and helpful comments; Carolyn DeLautre for manuscript preparation. This work was supported in part by the following: NIH R01 DK069766. This publication was also made possible by the Commonwealth of Kentucky Research Challenge Trust Fund; the W. M. Keck Foundation; The Jewish Hospital Foundation; and the Swiss National Science Foundation.


S. Ildstad has significant equity interest in Regenerex, LLC, a start-up biotech company based on the facilitating cell technology.


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Copyright information

© Springer Science+Business Media, LLC 2010

Authors and Affiliations

  • Yiming Huang
    • 1
  • Volker Enzmann
    • 2
  • Suzanne T. Ildstad
    • 1
  1. 1.Institute for Cellular TherapeuticsUniversity of LouisvilleLouisvilleUSA
  2. 2.Department of Ophthalmology, InselspitalUniversity of BernBernSwitzerland

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