PITTSBURGH, Nov. 5 (UPI) -- A new neural stem cell drug delivery system has been created by U.S. scientists to fight Hunter syndrome and ultimately a variety of genetic disorders.
Carnegie Mellon University researchers led by Assistant Professor Stefan Zappe and graduate student Sasha Bakhru are creating genetically engineered adult neural stem cells for delivery to patients' brains where they will produce an essential missing protein.
Zappe, working with Dr. Raymond Sekula at Pittsburgh's Allegheny General Hospital, developed cell-instructive microcapsules that contain neural stem cells. The caviar-sized microcapsules will be used for rapid manipulation of stem cells outside the body and for reliable delivery to the brain.
"We are particularly interested in targeting the brain because this area of the body is protected by the so-called blood-brain barrier that has been very difficult to penetrate with therapeutic enzymes that are usually injected into the patient's bloodstream," Zappe said.
Added Sekula, "Our technology and methodology also will likely have far-reaching implications for hundreds of other diseases of the central nervous system."
