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Eye Drops Deliver Gene Therapy for Brain Disorders

(National Institute of Biomedical Imaging and Bioengineering) A research team including NIBIB-funded scientists has developed a simplified approach for delivering and monitoring gene therapy for brain disorders.
The group used eye drops to deliver the gene for a growth factor called granulocyte colony stimulating factor (G-CSF) in a mouse model of brain ischemia, which refers to a lack of blood and hence, oxygen, to the brain. 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 therapy in animal models of stroke, Alzheimer's dementia, Parkinson's disorder, and ALS. The system also offers the intriguing possibility that acute brain injury may someday be treated by emergency medical workers through the simple delivery of eye drops carrying a therapeutic gene.
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