Tag: New England Journal of Medicine
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Experimental Gene Therapy Restores Immune Function in Children with ADA-SCID: A Breakthrough in Immunodeficiency Treatment
Overview: A new path for ADA-SCID treatment Researchers from UCLA, University College London, and Great Ormond Street Hospital have reported a remarkable milestone in the fight against ADA-SCID, a rare and often fatal genetic immune disorder. In a long-term study published in the New England Journal of Medicine, an experimental gene therapy restored and sustained…