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lüll Progress and prospects: gene therapy for inherited immunodeficiencies Qasim W; Gaspar HB; Thrasher AJGene Ther 2009[Nov]; 16 (11): 1285-91Haematopoietic 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.|Animals[MESH]|Gene Silencing[MESH]|Genetic Therapy/*methods/trends[MESH]|Humans[MESH]|Immunologic Deficiency Syndromes/*therapy[MESH]|Male[MESH]|Mutagenesis, Insertional[MESH]|Severe Combined Immunodeficiency/therapy[MESH]|Wiskott-Aldrich Syndrome/therapy[MESH] |