New therapies to reverse blindness on the anvil

Therapies to reverse blindness caused by common retinal diseases such as macular degeneration and retinitis pigmentosa could soon be on the anvil, thanks to cutting-edge stem cell technology developed to correct a genetic defect present in a rare blinding disorder.

A team of scientists from the University of Wisconsin-Madison used a recently developed technology to generate induced pluripotent stem (iPS) cells from a human patient with an uncommon inherited eye disease known as gyrate atrophy.

This disorder affects retinal pigment epithelium (RPE) cells, the cells critical to the support of the retina's photoreceptor cells, which function in the transmission of messages from the retina to parts of the brain that interpret images.

"When we generate iPS cells, correct the gene defect that is responsible for this disease, and guide these stem cells to become RPE cells, these RPE cells functioned normally," said lead study author cell biologist Jason Meyer, Ph.D., assistant professor of biology in the School of Science at Indiana University-Purdue University Indianapolis.

“This is exciting because it demonstrates we can fix something that is out of order. It also supports our belief that in the future, one might be able to use this approach for replacement of cells lost or malfunctioning due to other more common diseases of the retina," she added.

Researchers are hopeful that once the gene defect responsible for a blinding disorder is corrected in iPS cells, these cells may be able to restore vision.

The study appeared in an online publication of the journal Stem Cells.