![]() Schwartz and his team built a cell production facility at CHOC and his next step is to convert it into a FDA-compliant cell manufacturing operation, where one day the stem cells can be transplanted into children, providing them, for the first time, a comprehensive treatment that has the possibility of curing them. Schwartz uses Induced Pluripotent Stem Cell (iPSC) technology to transform skin stem cells to brain stem cells, allowing him to study brain disease without ever touching the brain. ![]() Schwartz seeks to gain a better understanding of other brain diseases including autism. Stem cells have the ability to self-renew. CHOC is the only hospital in the country with a focus on using immune-matched stem cells to treat enzyme deficiencies of the brain. Stem cells have unique abilities to self-renew and to recreate functional tissues. Without this enzyme, glycosaminoglycans build up and damage organs, including the brain. Individuals with this disease do not make an enzyme that helps break down long chains of sugar molecules called glycosaminoglycans. iPSC are derived from skin or blood cells that have been reprogrammed back into an embryonic-like pluripotent state. Children with this disease generally die before their 10th birthday. Adult stem cells (also known as somatic or tissue stem cells) are already specialized stem cells that can only form certain cell types of the human body. Schwartz’s starting point for treatment is Hurler’s Syndrome, a rare, inherited disease of metabolism called a lysosomal storage disorder. When stem cells divide, they can form more stem cells. Schwartz and his team use stem cell research not only to treat disease, but also to gain a better understanding of disease processes– working to unlock mysteries that will eventually become treatments and one day, cures.ĭr. A stem cell is a cell with the potential to form many of the different cell types found in the body. Schwartz, PhD, a senior scientist at CHOC and research biologist at UC Irvine, has been involved in pediatric brain research for 25 years. Using Stem Cells to Treat Pediatric Brain Disorders Cell Stem Cell is a broad-spectrum journal that covers the entire spectrum of stem cell biology.
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