Gaspar, HB; Thrasher, AJ; (2005) Gene therapy for severe combined immunodeficiencies. Expert Opin Biol Ther , 5 (9) 1175 - 1182. 10.1517/147125126.96.36.1995.
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Severe combined immune deficiencies (SCIDs) are a group of monogenic diseases resulting in profound disturbances of lymphocyte development and function. Affected individuals are prone to life-threatening infections and without treatment do not survive beyond the first year of life. Haematopoietic stem cell transplantation from a well-matched donor offers high rates of survival, but in the absence of a suitable matched donor, parental haploidentical transplants are associated with greater complications, lower success rates and in some instances poor long-term immune recovery. Alternative therapeutic options based on correction of the defective gene by retroviral gene delivery have been used to correct X-linked SCID (SCID-X1) and adenosine deaminase-deficient SCID (ADA-SCID). A number of clinical trials have established that ex vivo gene transfer into haematopoietic progenitor cells allows effective recovery of immune defects and that gene therapy can offer a successful alternative to transplantation. The development of leukaemia as a result of insertional mutagenesis in one trial of gene therapy for SCID-X1 has raised concerns regarding the toxicity of retroviral vector-based gene delivery. These side effects are now being studied in detail and measures to prevent such events through alternative vectors delivery systems are in development at present.
|Title:||Gene therapy for severe combined immunodeficiencies.|
|Keywords:||Adenosine Deaminase, Adolescent, Adult, Animals, Child, Preschool, Clinical Trials as Topic, Genetic Therapy, Genetic Vectors, Hematopoietic Stem Cell Transplantation, Hematopoietic Stem Cells, Humans, Infant, Interleukin Receptor Common gamma Subunit, Leukemia, Receptors, Interleukin, Retroviridae, Severe Combined Immunodeficiency, T-Lymphocytes, Transduction, Genetic|
|UCL classification:||UCL > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Child Health > Department of Infection and Immunity > ICH - Molecular Immunology Unit|
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