Tag: bone marrow transplant alternatives
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Portlaoise Boy’s Life-Changing ADA-SCID Gene Therapy Trial Results
Life-Changing Breakthrough for Portlaoise Boy in ADA-SCID Gene Therapy Trial A nine-year-old boy from Portlaoise, Ireland, has witnessed transformative improvements after participating in a landmark medical study targeting ADA-SCID, a rare and life-threatening immune system disorder. The trial, conducted in part at Great Ormond Street Hospital (GOSH) in London, represents a significant milestone in the…
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Experimental Gene Therapy Restores Immune Function in Children with ADA-SCID
Breakthrough in ADA-SCID treatment: Gene therapy shows lasting immune restoration Researchers have reported a landmark advance in treating ADA-SCID, a life-threatening genetic immune disorder. An experimental gene therapy developed by teams at UCLA, University College London (UCL), and Great Ormond Street Hospital (GOSH) restored and sustained immune system function in 59 of 62 children born…