Gene therapy for genetic haematological disorders and immunodeficiencies

Kohn DB (Children’s Hospital and Keck School of Medicine, Los Angeles, CA, USA). Gene therapy for genetic haematological disorders and immunodeficiencies. J Intern Med 2001; 249: 379–390. Gene transfer and autologous transplantation of haematopoietic stem cells (HSCs) from patients with genetic haematological disorders and immunodeficiencies could provide the same benefits as allogeneic HSC transplantation, without the attendant immunological complications. Inefficient gene delivery to human HSCs has imposed the major limitation to successful application of gene therapy. A recently reported clinical trial of gene transfer into HSCs of infants with X‐linked severe combined immunodeficiency (SCID) has achieved immune restoration because of the selective outgrowth of the gene‐corrected lymphocytes. Newer methods for manipulating HSCs may lead to efficacy for other disorders. The problems and progress in this area are reviewed herein.