1. Progress and prospects: gene therapy for inherited immunodeficiencies.
- Author
-
Qasim, W., Gaspar, H. B., and Thrasher, A. J.
- Subjects
- *
IMMUNODEFICIENCY , *GENE therapy , *CHRONIC granulomatous disease , *HEMATOPOIETIC stem cells , *ADENOSINE deaminase deficiency - 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. [ABSTRACT FROM AUTHOR]
- Published
- 2009
- Full Text
- View/download PDF