Philip K. Liu, Ph.D., of the Martinos Center for Biomedical Imaging at Harvard Medical School, and his collaborators, Drs. H. Prentice and J. Wu of Florida Atlantic University have developed a technique which quickly delivers and monitors gene therapy for brain disorders. The doctors used a simplified technique of eye drops to deliver the granulocyte colony stimulating factor into a mouse model of brain ischemia. Brain ischemia is a condition where there is insufficient blood flow to the brain.
The G-CSF was transported by inserting the gene into an adenovirus. The adenovirus was then administered through eye drops. The scientists concluded the growth factor treatment led to a reduction in brain atrophy, neurological defects, and the death in the mice. After the growth factor drops were added to the mice, MRI was used to monitor the success. The overall combination of non-invasive, simple delivery has the potential to improve experimental gene therapy in animal models.
The primary target for Dr. Liu and his team was to develop a simple technique that administers the G-CSF quickly to the brain without the accompaniment of highly trained staff and elaborate technologies. This expeditious process could be the difference in saving a stroke or cardiac arrest patient’s life. The second target for Dr. Liu was to include a noninvasive procedure that delivers G-CSF to the brain expressed at therapeutic levels. The target is to administer the system to animals then translate over to humans.
This simple technique could lead to the improvement of stroke, Alzheimer’s dementia, Parkinson’s disorder and ALS.
Although the test results in human trials were unsuccessful, I believe this gene therapy study is quite beneficial to the study of human health. This experiment could lead to the potentially life saving growth factor in critical patients. Because this process is noninvasive, it is also a lot less stressful on people with an illness.