Developing novel gene therapy technology for treatment of glycine encephalopathy (360G-Wellcome-210774_Z_18_Z)

"Glycine encephalopathy, also known as non-ketotic hyperglycinaemia (NKH), is a life limiting inherited neuro-metabolic disease which presents soon after birth and leads to severe neurological outcomes including epilepsy and profound developmental delay. Current treatments for NKH are neither effective long-term, nor curative. Glycine encephalopathy is characterized by accumulation of glycine and is known to result from mutation of genes that encode the glycine cleavage system so that glycine cannot be broken down. The majority of affected children carry mutations in GLDC, encoding glycine decarboxylase. In this project we aim to develop gene therapy for glycine encephalopathy to restore GLDC function in the liver using lentiviral vector to provide permanent delivery of the therapeutic sequence. In a glycine encephalopathy model we will test a novellentiviral vector that offers enhanced safety and performance. This will be a key step towards potential clinical implementation of this therapy