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Gene therapy for severe combined immunodeficiencies.
Expert Opin Biol Ther. 2005 Sep; 5(9):1175-82.EO

Abstract

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.

Authors+Show Affiliations

Institute of Child Health, 30 Guilford Street, London, WC1N 1EH, UK. h.gaspar@ich.ucl.ac.ukNo affiliation info available

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

16120048

Citation

Gaspar, H Bobby, and Adrian J. Thrasher. "Gene Therapy for Severe Combined Immunodeficiencies." Expert Opinion On Biological Therapy, vol. 5, no. 9, 2005, pp. 1175-82.
Gaspar HB, Thrasher AJ. Gene therapy for severe combined immunodeficiencies. Expert Opin Biol Ther. 2005;5(9):1175-82.
Gaspar, H. B., & Thrasher, A. J. (2005). Gene therapy for severe combined immunodeficiencies. Expert Opinion On Biological Therapy, 5(9), 1175-82.
Gaspar HB, Thrasher AJ. Gene Therapy for Severe Combined Immunodeficiencies. Expert Opin Biol Ther. 2005;5(9):1175-82. PubMed PMID: 16120048.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Gene therapy for severe combined immunodeficiencies. AU - Gaspar,H Bobby, AU - Thrasher,Adrian J, PY - 2005/8/27/pubmed PY - 2006/11/15/medline PY - 2005/8/27/entrez SP - 1175 EP - 82 JF - Expert opinion on biological therapy JO - Expert Opin Biol Ther VL - 5 IS - 9 N2 - 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. SN - 1744-7682 UR - https://www.unboundmedicine.com/medline/citation/16120048/Gene_therapy_for_severe_combined_immunodeficiencies_ L2 - http://www.tandfonline.com/doi/full/10.1517/14712598.5.9.1175 DB - PRIME DP - Unbound Medicine ER -