Gene Therapy Utilizing Modified Stem Cells Treats Rare Immune Disorder

Published 2025-05-06

A groundbreaking gene therapy trial has successfully treated a four-year-old boy with leukocyte adhesion deficiency 1 (LAD-1), a rare and often fatal immune disorder. The treatment involved extracting the patient's own hematopoietic stem cells, correcting the genetic defect using a lentiviral vector, and reintroducing the modified cells into his body. Post-treatment, the boy has shown a fully functioning immune system and is living a normal life. This approach not only offers a potential cure for LAD-1 but also exemplifies the therapeutic potential of combining gene therapy with autologous stem cell transplantation for treating genetic disorders.