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Recent Advances in Vision Science
National Eye Institute Science Advances

Induced Pluripotent Stem Cells (iPSC) Are Used to Prevent Vision Loss in Rodent Model of Retinal Degeneration


Retinal neurodegenerative diseases, such as age-related macular degeneration (AMD), are the leading cause of irreversible vision loss in older Americans. AMD involves degeneration and destruction of the retinal pigment epithelium (RPE), a thin layer of tissue that supports and nourishes the light-detecting photoreceptor cells in the neural retina. Replacing damaged RPE cells using normal, functioning RPE cells is not yet possible. However, the recent discovery that stem cells can be created from adult, rather than embryonic cells, provides a new approach to generate RPE cells for replacing damaged tissue. If proven therapeutically efficacious, these stem cells, known as human induced pluripotent stem cells (iPSC), could provide treatments for a variety of diseases thus avoiding ethical concerns regarding the need to work with human embryonic stem cells in research.


NIH investigators used human iPSC to create functional RPE cells in culture. These cells demonstrated normal RPE cell functions such as phagocytosis (digestion) of older photoreceptor cell material. After characterizing several physiological properties of the iPSC-derived RPE cells, the cells were transplanted into the eye of a rodent model of retinal degeneration caused by defective RPE. These new, transplanted RPE cells functioned normally and resulted in long-term preservation of vision in this rodent disease model.

Public Impact Statement/Significance:

This translational research effort leveraged an exciting basic science discovery, namely the generation of RPE cells from human iPSC, for use as donor tissue in cell replacement therapy for diseased tissue. The studies demonstrate the tremendous potential for iPSC in treating certain retinal degenerative diseases and set the stage for examining the behavior of iPSC in a variety of ocular disorders.

Grant Support:

NEI 5R24EY014799-05; NCRR 1S10RR017753-07.

Publication Citations:

Buckholz, D.E., et al. Derivation of Functional Retinal Pigmented Epithelium from Induced Pluripotent Stem Cells. Stem Cells. 2009; 27(10): 2427-34.

Carr, A.J., et al. Protective effects of human iPS-derived retinal pigment epithelium cell transplantation in the retinal dystrophic rat. PLoS One. 2009; 4(12): e8152. PubMed

Last Reviewed: January 2010

Department of Health and Human Services NIH, the National Institutes of Health