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"Breakthrough Gene Therapy Provides Decade-Long Immune Defense in Kids"
A groundbreaking gene therapy has shown lasting success in treating children with severe combined immunodeficiency by restoring their immune system function for over a decade.
- The experimental gene therapy was developed by teams at UCLA, University College London, and Great Ormond Street Hospital, effectively treating 59 of 62 children with adenosine deaminase deficiency.
- With a follow-up data totaling 474 years, five children have maintained healthy immune system function more than ten years post-treatment, showcasing the therapys long-term efficacy.
- This study marks a significant advancement over traditional hematopoietic stem cell transplantation, as it directly addresses the mutation causing severe combined immunodeficiency without the need for donor cells.
Why It Matters
This breakthrough in gene therapy signifies a potential paradigm shift in treating genetic disorders like severe combined immunodeficiency, offering hope to families affected by such life-threatening conditions. The long-term success of the treatment could inspire further research into similar therapies for other genetic diseases.