Gene Therapy Delivers Long-Term Immune Protection in Children With Rare Immune Disorders

Recent studies report that gene therapy has successfully delivered lasting immune protection for children with two rare and life-threatening immune disorders:
adenosine deaminase severe combined immunodeficiency (ADA-SCID) and severe leukocyte adhesion deficiency-I (LAD-I)2 4 9 13.

For ADA-SCID, the largest cohort to date (nearly 50 children) showed a 100% survival rate and over 95% cure rate with gene therapy; the therapy safely restored immune system function with effects lasting at least 2–3 years post-treatment2 7 13.

ADA-SCID is caused by mutations in the ADA gene, leading to a lack of immunity and high risk of fatal infections if untreated. Gene therapy corrects these mutations in a patient’s own stem cells, offering a one-time, curative procedure2 13.

For LAD-I, an international trial treated nine children aged 5 months to 9 years; all recovered immune function, resolved disease symptoms (such as skin lesions and gum inflammation), and were free of recurrent infections up to two years after treatment4 5 9.

LAD-I is an ultra-rare disorder caused by mutations affecting the CD18 protein, leaving patients highly susceptible to infections. Without treatment, most children do not survive past age two4 5 9.

This gene therapy approach involves genetically correcting the patient’s own blood stem cells, which helps avoid the complications of donor transplants such as graft-versus-host disease4 9.

The clinical trials were led by international teams including UCLA, Great Ormond Street Hospital (GOSH), and University College London (UCL), and the results were published in the New England Journal of Medicine2 5 9 13.