A recent international study followed 62 children treated between 2012 and 2019 for the rare immune-deficiency condition ADA‑SCID, using ex-vivo stem cell lentiviral gene therapy. After a mean follow-up of 7.5 years, the treated children had a 100% survival rate, and over 95% were considered cured. This research was conducted by University College London.
This is a remarkable outcome because many gene therapies for rare childhood conditions face challenges with durability, immune reactions, or long-term safety. From a genetics perspective, it highlights how delivering a corrected gene to hematopoietic stem cells can provide a durable therapeutic approach.
The study also raises several questions and implications. How will these long-term safety data influence regulatory approval and access for other rare-disease gene therapies? What kind of genetic monitoring will be necessary as these children grow into adulthood? In our genetics course context, this research demonstrates how modern gene therapies can provide lasting, life-changing results for pediatric patients, bridging the gap between experimental science and clinical care.
In summary, while gene therapy often feels like a futuristic and unrealistic promise, this study demonstrates concrete, long-lasting benefits for children. These results make the topic both timely and meaningful for genetics, medicine, and public communication.
Sources
University College London. (2025, October). Landmark gene therapy study shows safety in children. UCL News. https://www.ucl.ac.uk/news/2025/oct/landmark-gene-therapy-study-shows-safety-children
Poletti, M., & Mavilio, F. (2021). Designing lentiviral vectors for gene therapy of genetic diseases. PMC. https://pmc.ncbi.nlm.nih.gov/articles/PMC8402868/
You summarized this study really well, and I agree it’s an important milestone for gene therapy. The long-term success rate is amazing, especially since many treatments struggle with durability or immune complications. I like how you connected the corrected ADA gene in stem cells to broader questions about future regulation and monitoring. This really shows how genetics research can translate into real, life-changing clinical outcomes for children with rare diseases.
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