Genetically Modified Cells Migrate To Brain And Treat Neurodegeneration In St. Jude Model

Name: Joanne Tham (s4209695)


This article is about the effect of a genetically modified gene that has the ability to cure patients from a fatal disease known as Lysosomal Storage disease (LSDs), by being able to transport the desired drug to dying neural cells in the brain.

This cure was discovered by St.Jude researchers who were trying to treat a type of LSDs known as GM1--Gangliosidosis by using Bone Marrow Cells (BMC) and inserting an enzyme that breaks down fat molecules GM1. GM1 is an essential component in the brain, however patients who are suffering from GM1--Gangliosidosis lack the enzyme beta-glycosidase. This results in an increased concentration of GM1 which damages the cell and causes it to die. The function of BMC is that it produces a wide variety of cells with different functions, one example being the immune cells known as monocytes. This gene was then genetically modified and inserted into the a laboratory model. The results showed that the monocytes migrated toward the site lacking in beta-glycosidase, the enzymes released were then used by the cells to break down excess GM1 which help to prevent the buildup of more GM1.

The migration of the monocytes were because they followed the gradient-increasing concentration signals being produced by the cells adjacent to the dying neurons known as chemokines until they reached the dying neurons. In normal circumstances, the immune cells would cause greater problems to the neurons as they made them became inflamed. However, because these cells were genetically modified monocytes, it helped to restore the beta-glycosidase activity which reduced the levels of dying neurons as well as chemokines.

In conclusion, if the BMCs were able to be successfully modified, this would be an effective form of therapy. The genetic modification of the BMCs would be able to produce unlimited amounts of the modified monocytes, hence ensuring that there would always be sufficient enzymes to reduce the activity of beta-glycosidase. In addition, if the cells were able to be replicated by the same patient after they were modified, this would also help to get rid of complications in finding a suitable donor.


Reference List:

St. Jude Children's Research Hospital (2005) “Genetically Modified Cells Migrate To Brain And Treat Neurodegeneration In St. Jude Model” Available at: http://www.sciencedaily.com/releases/2005/07/050718005942.htm Viewed on 12/5/09