The article shares compelling patient stories, including that of Cora Oakley, born without an immune system and once at risk from even a common cold. Gene therapy trials that correct the genetic defect in a child’s own stem cells have yielded exceptional results: in a cohort of 62 infants with ADA-SCID, all survived long-term and nearly all achieved full immune reconstitution.
Reflecting on the field’s evolution, Prof. Staal noted, “This was a fairy tale 20 years ago. Now it’s a reality.” His words capture the remarkable scientific progress and the sustained effort that made it possible.
At LUMC, Prof. Staal’s group has been at the forefront of developing safe and effective gene therapies for immune deficiencies, advancing vector technologies and translational strategies that now benefit patients worldwide. The recognition in The New York Times highlights both the clinical impact of these therapies and the importance of long-term commitment to innovation.
As gene therapy broadens to more SCID subtypes and inherited immune disorders, Prof. Staal’s contributions will continue to shape the field’s future.
These achievements demonstrate the strength of translational immunology at LUMC. We are especially proud that such a strategic and visionary research group, led by Prof. Staal, is part of the IMMU Department, driving innovations that define the future of immune medicine. Please read the article here.