Our clinical scientist Roly Megaw has just been awarded a Wellcome Trust Seed Award for our exciting blue sky science project, entitled ‘In vivo genome editing of post-mitotic mammalian photoreceptors for treatment of genetic vision loss’.
Retinitis Pigmentosa (RP) is caused by ‘spelling mistakes’ in our DNA. These spelling mistakes, called 'mutations', result in death of the light-sensing cells in our retina, causing blindness. At present, we have no treatment for RP.
A potential breakthrough for genetic disease has been the discovery of CRISPR genome editing. This involves small ‘gene scissors’ that can be programmed to precisely cut DNA. Using this, scientists have shown we can replace the misspelt, disease-causing section of a gene by ‘pasting’ in a correct, healthy copy in its place.
In theory, ‘gene repair’ by CRISPR technology could be performed in any cell type, offering a treatment for all genetic diseases. If the gene repair could be applied to the eye's light-sensing cells, this could include RP. This project will address key obstacles to making this a reality. We hope genome editing technology can be developed as a treatment for all RP-causing mutations.