Retinal Restoration

There’s currently no cure for retinitis pigmentosa (RP), a group of inherited eye diseases that impair the retina’s ability to respond to light, resulting in gradual loss of vision. But now, researchers have used stem cells to develop a promising experimental treatment. First they reprogrammed skin cells from RP patients into stem cells to make patient-specific retinal cells for closer inspection. They found that mutations in a gene called MFRP disrupt the production of actin (red), a protein that provides scaffolding for retinal cells. When this structure doesn’t form properly (left), retinal cells don’t work very well. But when the team used a virus to smuggle in a working copy of MFRP, the structure was restored (right). And in mice with an RP-like condition, the treatment slowly improved vision. It's early days yet but these results show how patient-specific stem cells can kick start the development of tailor-made therapies.

Written by Daniel Cossins

Daniel Cossins

Writer

As a freelance science writer, Dan's work has been published in award-winning magazines including BBC Focus, Wired UK, Cosmos, BBC Knowledge, BBC History and Men's Health.

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• Image by Stephen Tsang and colleagues
• Columbia University Medical Center, USA
• Originally published under a Creative Commons Licence (BY 4.0)
• Research published in Molecular Therapy, July 2014