Eye drops deliver gene therapy for brain disorders

Eye drops have been used to deliver the gene for a growth factor called granulocyte colony stimulating factor (G-CSF) in a mouse model of brain ischemia. The treatment led to a significant reduction in brain atrophy, neurological deficits, and death in the mice. The research team also devised a system to monitor the success of the gene delivery using MRI. The combination of simple delivery and non-invasive monitoring has the potential to contribute to improved studies of experimental gene therap