22 July 2014
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