Tag: GOSH
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ADA-SCID Gene Therapy Shows Long-Term Success in Children
Breakthrough in pediatric ADA-SCID treatment A large international study led by researchers from Great Ormond Street Hospital (GOSH), University College London (UCL), and the University of California, Los Angeles (UCLA) has demonstrated the long-term safety and effectiveness of a standard lentiviral gene therapy for ADA-SCID, a rare and life-threatening immune disorder. The cohort, followed for…
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Experimental gene therapy restores immune system function in ADA-SCID children
Groundbreaking therapy offers durable immune restoration for ADA-SCID An experimental gene therapy developed by researchers at UCLA, University College London (UCL) and Great Ormond Street Hospital (GOSH) has achieved durable restoration of immune system function in 59 of 62 children born with ADA-SCID, a rare and often deadly genetic immune disorder. The long-term follow-up results,…
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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…
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Gene Therapy Shows Long-Term Success for Children with ADA-SCID
Groundbreaking Study Highlights Long-Term Success of ADA-SCID Gene Therapy A landmark international study led by researchers from Great Ormond Street Hospital (GOSH), University College London (UCL), and the University of California, Los Angeles (UCLA) shows that gene therapy can provide durable, life-changing results for children with ADA-SCID, a rare and life-threatening immune deficiency. The team…