AANS Neurosurgeon | Volume 26, Number 1, 2017

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Eye Drops Deliver Gene Therapy for Brain Disorders

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A research team recently developed a simplified approach for delivering and monitoring gene therapy for brain disorders. The group, from the National Institute of Biomedical Imaging and Bioengineering (NIBIB), 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. 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 amyotrophic lateral sclerosis (ALS). The system also offers the possibility that acute brain injury may someday be treated by emergency medical workers through the simple delivery of eye drops carrying a therapeutic gene. This new, rapid, non-invasive administration and evaluation of gene therapy has the potential to be successfully translated to humans,” said the director of the NIBIB Division of Applied Science and Technology. “The use of MRI to specifically image and verify gene expression, now gives us a much clearer picture of how effective the gene therapy is. The dramatic reduction in brain atrophy in mice, if verified in humans, could lead to highly effective emergency treatments for stroke and other diseases that often cause brain damage such as heart attack.” A main goal of the research team’s is to develop a simplified technique that would allow rapid delivery of G-CSF to the brain without elaborate technologies requiring highly trained staff and equipment, particularly important for stroke and cardiac arrest. To read more about this study, click here.

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