Tag: lentiviral


  • ADA-SCID Gene Therapy Shows Long-Term Success in Children

    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…

  • Long-term success for gene therapy in children with ADA-SCID: a landmark study

    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…

  • Gene Therapy Shows Long-Term Success for Children with ADA-SCID

    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…