Safety and efficacy of perinatal gene therapy for Neuronopathic Gaucher disease

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Gaucher disease is a genetic disease which, in its severe form, results in serious brain damage infant death before two years of age. The disease is incurable with currently-available drugs since they can?t enter the nerves in the brain. Gene therapy has already been used to cure several genetic diseases and, very recently, it has become possible to deliver genes to the brain. Based on this recent finding, we are trying to cure a mouse model of the human brain disease using gene therapy. We will be injecting the gene into bloodstream of fetal and neonatal mice to see if this prevents the onset of brain disease. If this works it may provide a pathway towards treating human beings; at the very least we will learn more about why the disease damages the brain in infants.