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6. Medical College of Georgia’s Research Institute, Robert K. Yu Dr. Robert Yu and his team at the Medical College of Georgia (MCG) have engaged in developing a cell-transplantation therapy to alleviate the devastating effects of Sanfilippo disease (mucopolysaccharidosis III). The stem cells are derived from the inner cell mass in the blastocytes at early embryonic stages of development. Recent studies indicate stem cells can also be isolated from other sources, such as umbilical cord blood, amniotic fluid, and even adipose tissue. These primitive stem cells are self-renewable and multipotential, meaning they can develop into different cell types, such as nerve, muscle, and bone. For this reason, stem cells can be harvested and transplanted into damaged brain, where they then can repopulate damaged tissues. Transplantation therapy, therefore, represents a viable therapeutic approach for repairing injured brain tissue caused by many different neurogenerative diseases, including Sanfilippo disease, multiple sclerosis, strokes, Parkinson’s disease, and so on. For practical purposes, Dr. Yu has induced stem cells to differentiate into neural stem cells (NSCs) that are committed to developing into brain cells. Once these cells are transplanted into damaged brain, they can correct some of the metabolic defects of Sanfilippo disease (MPSIII). During the past year, Dr. Yu and his team have learned much about the properties of NSCs and how they populate and interact properly with each other in test tubes. This basic knowledge is essential because Dr. Yu wishes to make sure that he can produce enough cells for transplantation. Once the cells are transplanted, he wants to make sure that they survive, migrate, and grow normally within the recipient’s brain, and also that they avoid abnormal growth (e.g., tumor growth). Dr. Yu and his team have already transplanted these cells into the brain of animal models with MPSIII to see if these cells can correct the storage of heparan sulfate. Preliminary studies have shown that transplanted NSCs grow after transplantation. More important, the recipient animals survive the surgery for a prolonged time. Animals with MPS appear to have improved biological function, as assessed by behavioral tests, which is very encouraging. Embryonic stem cells (ECSs) may also be amenable to ex vivo engineering by DNA modifications that render them more suitable for gene therapy. Using viral infection, Dr. Yu has successfully fortified ESCs with enzymes that are missing in MPS cells. He plans to transplant these engineered cells into animal brain with MPS in an attempt to restore function in the diseased tissue. He is keenly aware that the safety and efficacy of these strategies must be fully evaluated before their use in humans with MPS. In preparation for use in humans, Dr. Yu has established the first state-supported umbilical cord blood bank in the state of Georgia. Umbilical cord blood is rich in stem cells that can be harvested for clinical use. The bank is supported by funds provided by Georgia and by MCG, among other sources. In fact, the governor of Georgia, Sonny Purdue, has signed a bill establishing the Commission of Cord Blood Stem Cell Research and Medical Treatment and has appointed Dr. Yu as a member of the commission. Dr. Yu and his team anticipate having sufficient numbers of stem cells in the near future to investigate the treatment of a variety of neurodegenerative conditions, including Sanfilippo disease. |
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