Gene therapy for leukocyte adhesion deficiency.

摘要: Abstract Leukocyte adhesion deficiency (LAD) is an autosomal recessive immunodeficiency disease characterized by severe, recurrent bacterial infections. In patients with LAD, the leukocytes, particularly neutrophils, fail to adhere endothelial cell wall and migrate site of infection. LAD results from heterogeneous molecular defects in leukocyte integrin CD18, which prevent CD11/CD18 heterodimer formation surface expression. To date, hematopoietic stem transplantation remains only curative treatment for however, this approach limited transplant-related toxicities graft-versus-host disease. During course preceding decade we have conducted extensive experimental studies demonstrating that gene transfer CD18 subunit corrects structural functional defect leukocytes. These provided support initiation a clinical trial retroviral-mediated two severe phenotype or LAD. This review will present overview preclinical initial current trial.