Tag: pediatric immunology
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Landmark ADA-SCID gene therapy delivers life-changing results for Portlaoise boy
Overview: a breakthrough in ADA-SCID treatment A Portlaoise boy’s participation in a landmark medical study has yielded life-changing results, offering new hope for children born with the rare immune condition ADA-SCID. The nine-year-old, Andy Cash, was diagnosed with the life-threatening disorder just weeks after birth, and his family faced a long, difficult journey before a…
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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…