Carnegie Mellon researchers to develop new drug deliv...( PITTSBURGH Carnegie Mellon Universi...)

PITTSBURGH Carnegie Mellon Universitys Stefan F. Zappe is using adult neural stem cells to develop a new stem cell-based drug delivery therapy that may ultimately help treat a variety of inherited genetic disorders like Hunter syndrome.

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 patients 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.

Zappe will be using these caviar-sized capsules specifically for rapid manipulation of stem cells outside the body and for reliable delivery of stem cells to the brain. The acute inflammatory response that usually occurs from implantation would normally cause implanted neural stem cells to differentiate into mature cell types that are not able to migrate extensively. Encapsulated stem cells will be protected fr
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