A New Source of Cells for Treating Blindness

An article published in Experimental Biology and Medicine (Volume 246, Issue 2, January, 2021) (https://journals.sagepub.com/doi/pdf/10.1177/1535370220963755) reports a cell replacement therapy for blindness caused by loss of photoreceptors. The study, led by Dr. Jian Feng, Professor of Physiology and Biophysics, State University of New York at Buffalo in New York (USA), demonstrates that adult human retinal pigment cells can be converted to neurons that exhibit many properties of photoreceptors.
Photoreceptor degeneration is a leading cause of blindness. Photoreceptors are specialized neurons in the retina that allow us to see by converting the information in light into signals for transmission to the brain. Because photoreceptors are neurons, the body cannot generate new photoreceptors after injury. One alternative is transplantation of photoreceptors generated in a dish into the retina. Human retinal pigment epithelial cells are readily available from organ donors and originate from the same structure during development as photoreceptors. While their proliferative potential and ability to transdifferentiate into other cell types make them an attractive source, there are no established methods for converting retinal pigment epithelial cells into photoreceptors.
In this study, Feng and colleagues examined the ability of the transcription factor ASCL1 and gene expression regulator miRNA124 to convert human retinal pigment epithelial cells to neurons. After 10 days of treatment, retinal pigment epithelial cells exhibited many properties of photoreceptors including expression of photoreceptor-specific genes and production of membrane stacks similar to photoreceptor discs. Although the converted neurons did not respond to light stimulation, this study paves the way for further developments that may generate light-responsive photoreceptors suitable for transplantation. Dr. Feng said, “It is quite remarkable that adult human retinal pigment epithelial cells from donated retinas can be used to make neurons with many properties of photoreceptors. This promising method may eventually produce functional photoreceptors that can be used to cure blindness.”
Dr. Steven R. Goodman, Editor-in-Chief of Experimental Biology & Medicine, said, “Dr. Feng and colleagues have taken a major step towards a cell-based therapy for photoreceptor degeneration that leads to blindness, in being able to convert primary adult human retinal pigmented epithelium (hRPE) into post mitotic neurons with several properties of photoreceptors. Future studies will focus creating photoreceptors with all components required for light sensitivity.”
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Source: Experimental Biology and Medicine