Finding and treating the cause of inherited blindness
European scientists have made a major breakthrough in the treatment of inherited retinal diseases. Many disorders which cause blindness are due to mutations in genes that are expressed in the photoreceptor cells of the retina which process light. The European AAVEYE ('Gene Therapy for Inherited Severe Photoreceptor Diseases') project set out to develop gene transfer techniques for the photoreceptor neurons in the retina using a virus (adeno-associated virus, AAV) as a form of transport. The team focused on pre-clinical proof-of-concept (POC) gene therapy strategies to tackle these debilitating diseases.