Tag: lentiviral
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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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Long-term success for gene therapy in children with ADA-SCID: a landmark study
Groundbreaking long-term results for ADA-SCID gene therapy A multinational team led by Great Ormond Street Hospital (GOSH), University College London (UCL), and the University of California, Los Angeles (UCLA) has reported the longest follow-up to date for children treated with a gene therapy for ADA-SCID, a rare immune deficiency. The study, published in the New…
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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…