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6. Medical College of Georgia’s Research Institute, Robert K. Yu Dr. Yu and his team have continued their effort in developing a cell transplantation therapy to alleviate the devastating effects of Sanfilippo disease (Mucopolysaccharidosis III, MPSIII). Toward this end, they have employed neural stem cells (NSCs) for transplantation. There have been strong efforts worldwide in promoting these cells for the treatment of a variety of neurodegenerative diseases because stem cells are capable of becoming neural cells and integrate into the damaged brains once transplanted. One major uncertainty facing investigators in the field, however, is to ensure that these cells behave normally and do not turn into malignant cells. During the past year, Dr. Yu and his team have learned much about the properties of the NSCs in test tubes. This basic knowledge is essential because Dr. Yu needs to harvest enough corrective cells for transplantation. Once the cells are transplanted, he wants to make sure the donor cells survive, migrate, and grow normally within the recipient’s brain, and to avoid abnormal growth of these cells, e.g., tumor growth. Dr. Yu and his collaborators have already transplanted these cells into the brain of animal models with MPSIII to see if they can correct the storage of heparan sulfate. Preliminary studies have shown that these cells grow after transplantation. More importantly the recipient animals survive the transplantation surgery for a prolonged time. Animals with MPS appear to have improved biological function, as assessed by behavioral tests, which is very encouraging. Although there have been attempts at using gene therapy for MPSIII, there have been mixed results, especially when virus-based techniques are required. Several reports have appeared on the appearance of serious side effects. During the past year, Dr Yu and his team have started to explore a new technology for gene therapy, the so-called “Sleeping Beauty” or “transposon” technology. This is a novel way to replace missing or defective genes into host chromosomes. This technique is gaining momentum in the gene therapy field because of few side effects and the ability to integrate the missing genes into the host chromosomes. Embryonic stem (ES) cells may also be amenable to ex vivo engineering by DNA modifications that render them more suitable for gene therapy. Preliminary results from Dr. Yu’s laboratory have shown that it is feasible to fortify ES cells with enzymes that are missing in MPSIII using this technique. He plans to transplant these engineered cells into animal brain 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 MPSIII. In preparation for use in humans, Dr. Yu has established the first state-supported Umbilical Cord Blood Stem Cell Bank in the State of Georgia. Cord blood is rich in stem cells that can be harvested for clinical use. This bank is supported by funds provided by the State of Georgia and the Medical College of Georgia. In fact, the State of Georgia has reaffirmed its commitment to cord blood stem cell collection and research by appropriating additional funds to support this facility for 2009. In 2007, Governor Sonny Perdue signed a bill establishing the Commission of Cord Blood Stem Cell Research and Medical Treatment and appointed Dr. Yu as a member of the Commission. The Commission delivered its recommendation in December. To continue this effort, Governor Perdue has reappointed Dr. Yu as one of the members of the Commission of Saving the Cure in April, 2008. The purpose of this Commission is to provide guidance on state support for clinical and basic research in stem cells in Georgia. We anticipate that we will have sufficient stem cells for the treatment of a variety of diseases, including Sanfilippo disease, in the near future. |
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