The university of Glasgow Caledonian, in conjunction with Yorkhill Children's Charity are beginning work on researching possible drugs for the treatment of Leber's Congenital Amaurosis in young people. Through gene therapy and the lead doctors work with zebrafish, the hope is that sufferers of the disease will in the future be able to experience restored visual function.
Leber's Congenital Amaurosis is a rare eye condition, inherited from a parent, that appears in roughly 1 in every 80,000 people around the time of birth or shortly afterwards. The presentation of the condition can vary due its association with multiple genes, however, it is generally described as causing vision loss, without an associated lesion. Symptoms of LCA can normally be recognised as slow or poor pupillary response, nystagmus and severe to complete vision loss. One particular form of the condition was successfully treated through the use of gene therapy in 2008.
Dr Xinhua Shu will lead the research into the drug development programme following a £40,000 award. The projects direction will focus upon decreasing oxidative damage and improving light sensitive cells survival rate. This will be in conjunction with gene therapy treatment which in the long term, is hoped to offer restored visual function.
As a background, Dr Shu has worked with zebrafish during research projects, due to the parallels between the human retina, and that of the fish. Analysis of the role gypenosides play in protecting the cells of the retina will be an important part of the programme.