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Progress and prospects: gene therapy for inherited immunodeficiencies.
Gene Ther. 2009 Nov; 16(11):1285-91.GT

Abstract

Haematopoietic stem cell transplantation (HSCT) is now widely used to treat primary immunodeficiencies (PID). For patients with specific disorders (severe combined immunodeficiency (SCID)-X1, adenosine deaminase deficiency (ADA)-SCID, X-chronic granulomatous disease (CGD) and Wiskott-Aldrich Syndrome (WAS)) who lack a suitable human leukocyte antigen (HLA)-matched donor, gene therapy has offered an important alternative treatment option. The success of gene therapy can be attributed, in part, to the selective advantage offered to gene-corrected cells, the avoidance of graft-versus-host disease and to the use of pre-conditioning in patients with chemotherapy to facilitate engraftment of corrected cells. Adverse events have been encountered and this has led to detailed characterization of retroviral vector integration profiles. A new generation of self-inactivating retroviral and lentiviral vectors have been designed to address these safety concerns, and are at an advanced stage of preparation for the next phase of clinical testing.

Authors+Show Affiliations

Molecular Immunology Unit, UCL Institute of Child Health, London, UK.No affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

19776764

Citation

Qasim, W, et al. "Progress and Prospects: Gene Therapy for Inherited Immunodeficiencies." Gene Therapy, vol. 16, no. 11, 2009, pp. 1285-91.
Qasim W, Gaspar HB, Thrasher AJ. Progress and prospects: gene therapy for inherited immunodeficiencies. Gene Ther. 2009;16(11):1285-91.
Qasim, W., Gaspar, H. B., & Thrasher, A. J. (2009). Progress and prospects: gene therapy for inherited immunodeficiencies. Gene Therapy, 16(11), 1285-91. https://doi.org/10.1038/gt.2009.127
Qasim W, Gaspar HB, Thrasher AJ. Progress and Prospects: Gene Therapy for Inherited Immunodeficiencies. Gene Ther. 2009;16(11):1285-91. PubMed PMID: 19776764.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Progress and prospects: gene therapy for inherited immunodeficiencies. AU - Qasim,W, AU - Gaspar,H B, AU - Thrasher,A J, Y1 - 2009/09/24/ PY - 2009/9/25/entrez PY - 2009/9/25/pubmed PY - 2010/6/30/medline SP - 1285 EP - 91 JF - Gene therapy JO - Gene Ther. VL - 16 IS - 11 N2 - Haematopoietic stem cell transplantation (HSCT) is now widely used to treat primary immunodeficiencies (PID). For patients with specific disorders (severe combined immunodeficiency (SCID)-X1, adenosine deaminase deficiency (ADA)-SCID, X-chronic granulomatous disease (CGD) and Wiskott-Aldrich Syndrome (WAS)) who lack a suitable human leukocyte antigen (HLA)-matched donor, gene therapy has offered an important alternative treatment option. The success of gene therapy can be attributed, in part, to the selective advantage offered to gene-corrected cells, the avoidance of graft-versus-host disease and to the use of pre-conditioning in patients with chemotherapy to facilitate engraftment of corrected cells. Adverse events have been encountered and this has led to detailed characterization of retroviral vector integration profiles. A new generation of self-inactivating retroviral and lentiviral vectors have been designed to address these safety concerns, and are at an advanced stage of preparation for the next phase of clinical testing. SN - 1476-5462 UR - https://www.unboundmedicine.com/medline/citation/19776764/Progress_and_prospects:_gene_therapy_for_inherited_immunodeficiencies_ L2 - http://dx.doi.org/10.1038/gt.2009.127 DB - PRIME DP - Unbound Medicine ER -