ORANGE, Calif.--(BUSINESS WIRE)-- A CHOC Childrens research project, under the direction of Philip H. Schwartz, Ph.D., senior scientist at the CHOC Childrens Research Institute and managing director of the facilitys National Human Neural Stem Cell Resource, has been awarded a $5.5 million grant from the California Institute for Regenerative Medicine (CIRM). The grant will be used to develop a stem cell-based therapy for the treatment of mucopolysaccharidosis (MPS I), a fatal metabolic disease that causes neurodegeneration, as well as defects in other major organ systems. Based on a number of medical and experimental observations, children with inherited degenerative diseases of the brain are expected to be among the first to benefit from novel approaches based on stem cell therapy (SCT). Dr. Schwartz explains, While uncommon, pediatric genetic neurodegenerative diseases account for a large burden of mortality and morbidity in young children. Hematopoietic (bone marrow) stem cell transplant (HSCT) can improve some non-neural symptoms of these diseases, but does not treat the deadly neurodegenerative process. Our approach targeting the effects of the disease on organs besides the brain with HSCT and neurodegeneration with a second stem cell therapy specifically designed to treat the brain is a strategy for whole-body treatment of … Continue reading
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