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.
Very interesting article its nice to see that there is being progress made in terms of gene therapy and working on ways to inhibit these disorder by inserting healthy genes to do the job. Again, this process was tested in animals and has still yet to be effective among humans. The difficulties arrive when trying to create the drug so that is is safe to use among humans.
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