Rescuing mini retinas from eye disease with gene therapy

A new gene therapy approach holds promise for treating a common group of inherited eye disease, according to a new UCL and Trinity College Dublin study in lab-grown 'mini retinas'. The findings, published in Stem Cell Reports , may contribute to new treatments for retinitis pigmentosa (RP), which affects thousands of people worldwide. RP is a group of rare genetic disorders that involve a breakdown and loss of cells in the retina, which is the light sensitive tissue that lines the back of the eye. Common early stage symptoms include difficulty seeing at night and a loss of side (peripheral) vision, with blindness often developing in time. Scientists have known for some time that mutations in the gene 'RP2', which is responsible for making a protein essential for normal vision, are associated with RP diseases. However, there are currently no therapies to treat people living with a number of RP diseases. The research team used a modified common virus to deliver a normal, functioning copy of the RP2 gene into "mini retinas", which had been engineered from stem cells and which contained the defective version of the gene.