New Hope in the treatment of Hunter Syndrome
Posted by Admin on Nov 4, 2007
Researchers at Carnegie Mellon University developing a new stem cell-based drug delivery therapy using adult neural stem cells,that may ultimately help treat a variety of inherited genetic disorders like Hunter syndrome.
Hunter syndrome is a hereditary disease in which the breakdown of a mucopolysaccharide (a chemical that is widely distributed in the body outside of cells) is defective. This chemical builds up and causes a characteristic facial appearance, abnormal function of multiple organs, and in severe cases, early death.
Zappe, an assistant professor of biomedical engineering at Carnegie Mellon, and his graduate student Sasha Bakhru, are creating genetically engineered adult neural stem cells for delivery to patients’ brains, where they will be programmed to produce an essential missing protein. In Hunter syndrome, for example, patients are lacking the enzyme iduronate-2-sulfatase that helps cells break down certain waste products. One in every 130,000 boys is born with the rare but deadly genetic disorder.
Zappe, who is working with Dr. Raymond Sekula, a neurosurgeon at Allegheny General Hospital, said he selected adult neural stem cells for his work because they can be harvested from a patient’s brain, have the potential to be multiplied outside of the body, can be genetically engineered, can disperse within the brain once re-implanted and can replace all major cell types of the brain.
To support their therapeutic goals, Zappe and his team have developed cell-instructive microcapsules that contain neural stem cells. These microcapsules efficiently control whether stem cells proliferate (multiply), differentiate into more specialized cell types like neurons and to what extent implanted stem cells will be allowed to migrate to the host tissue.
The Details About the Research can be found at medisyne.com