Developing novel gene therapy technology for treatment of glycine encephalopathy

Glycine encephalopathy, also known as non-ketotic hyperglycinaemia, is a life-limiting inherited neuro-metabolic disease which presents soon after birth and leads to severe neurological problems including epilepsy and profound developmental delay. It is characterised by the accumulation of glycine resulting from the mutation of genes that encode the glycine cleavage system which is responsible for breaking down glycine. Most affected children carry mutations that affect the production of the enzyme glycine decarboxylase (GLDC). Current treatments for glycine encephalopathy are neither effective long-term, nor curative.

We will develop gene therapy for glycine encephalopathy that restores GLDC function in the liver using lentiviral vector to provide permanent delivery of the therapeutic sequence. We will test a novel lentiviral vector in a glycine encephalopathy model that offers enhanced safety and performance. This will be a key step towards potential clinical use of this therapy.