Gene therapy for genetic haematological disorders and immunodeficiencies.
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.
Kohn
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DB
Division of Research Immunology/Bone Marrow Transplantation, Children's Hospital, Los Angeles, CA, USA. dkohn@chla.usc.edu
D000243:Adenosine Deaminase / Q000235:genetics; D000818:Animals; D005199:Fanconi Anemia / Q000235:genetics / Q000628:therapy; D005776:Gaucher Disease / Q000235:genetics / Q000628:therapy; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D006402:Hematologic Diseases / Q000628:therapy*; D019337:Hematologic Neoplasms / Q000628:therapy; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D016086:Lentivirus; D012190:Retroviridae; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy; D014161:Transduction, Genetic
D000243:Adenosine Deaminase