Can gene therapy treat CLN5 Batten disease?
£50,000 over 12 months, (March 2016-May 2017)
This grant is administered and managed for the BDFA by Sparks, Children’s Medical Research Charity.
Professor Jonathan Cooper aims to determine whether gene therapy could be used to treat CLN5 disease Batten disease. It is thought that one potential way of treating Batten disease is gene therapy. Gene therapy works by introducing a healthy copy of a gene to cells so they can then make a functioning copy of the protein that is missing. A harmless virus is often used to carry the copy of the gene into the cell. For one form of Batten disease, CLN2 Batten disease, gene therapy is currently being tested in a clinical trial. It is thought that gene therapy may also be beneficial for other forms of the disease.
Who is involved?
Professor Jonathan Cooper (pictured below) is going to investigate whether gene therapy could be used to treat a different form of Batten disease – CLN5 Batten disease building on a successful long standing collaboration with Professor David Palmer and his group at Lincoln University, New Zealand. They have been working on using gene therapy in animal model of CLN5 Batten disease and the aim of this research project will seek to determine if the treatment can prevent the death of brain cells and if there are any unintended consequences that might be harmful in the long term. Ana Assis was appointed in 2016 as a Research Assistant based at Kings College, London, for this 1-year project. PSDL Lab members (far right-Ana Assis)