“Between all three clinical trials, 50 patients were treated, and the overall results were very encouraging,” stated Donald Kohn, MD (above), professor of microbiology, immunology, and molecular genetics, and researcher at University of California, Los Angeles (UCLA), who studies precision medicine therapy for severe combined immunodeficiency (SCID). These cells were then returned to the child where they could start producing normal immune system cells if the therapy was effective. The goal was to change the function of these stem cells to create the functions that they were missing. The researchers used the genetic technologies they had developed to deliver a healthy version of the gene that was defective in these children. These stem cells, in healthy individuals, would create the immune cells needed to avoid infection and stay healthy. The team enrolled 50 children into their clinical trials and collected blood-forming stem cells from each child. The researchers, co-led by Donald Kohn, MD, of UCLA and Claire Booth, PhD, of GOSH, applied a gene treatment that specifically targeted this unique cause of SCID. Gene Therapy for SCID Instead of Bone Marrow Transplantīetween 20, a team of researchers from University of California, Los Angeles (UCLA) and Great Ormond Street Hospital (GOSH) in London used precision medicine to treat a specific genetic cause of SCID, sometimes referred to as SCID due to adenosine deaminase deficiency, or ADA-SCID. It is believed that those who receive a transplant before the age of 3.5 months are most likely to survive SCID, regardless of the type of stem cell donor used. Even if successful, bone marrow transplants can lead to rejection of the transplant, causing long-term health problems and decreasing survival and quality of life. SCID is also treated by using a bone marrow transplant, a dangerous and complex procedure. Treatment for SCID involves recognizing infections as they begin and treating them promptly. While more than a dozen genes have been implicated in SCID, more than 80% of SCID infants do not have a family history of the condition, according to the National Institutes of Health (NIH), which funds SCID studies and developed the original newborn screening test. Severe combined immunodeficiency (SCID) is a rare genetic disorder that causes severe immune system effects and makes children born with this condition severely susceptible to infection. New genetic technology may lead to cures for many genetic diseases, starting with immune system disordersĪ new, experimental precision medicine treatment has developed a potential cure for a deadly immune disease that is fatal in the first two years of life if untreated.
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