Progress and prospects: gene therapy for inherited immunodeficiencies.

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: W Qasim, H B Gaspar, A J Thrasher
• Journal: Gene therapy (Gene Ther) Vol. 16 Issue 11 Pg. 1285-91 (Nov 2009) ISSN: 1476-5462 [Electronic] England
• PMID: 19776764 (Publication Type: Journal Article, Review)

Topics

• Animals
• Gene Silencing
• Genetic Therapy (methods, trends)
• Humans
• Immunologic Deficiency Syndromes (therapy)
• Male
• Mutagenesis, Insertional
• Severe Combined Immunodeficiency (therapy)
• Wiskott-Aldrich Syndrome (therapy)