Back to Journals » Stem Cells and Cloning: Advances and Applications » Volume 3

Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency

Authors Montiel-Equihua C, Thrasher A, Gaspar B

Published 22 December 2009 Volume 2010:3 Pages 1—12

DOI https://doi.org/10.2147/SCCAA.S5570

Review by Single anonymous peer review

Peer reviewer comments 2



Claudia A Montiel-Equihua, Adrian J Thrasher, H Bobby Gaspar

Centre for Immunodeficiency, Molecular Immunology Unit, UCL Institute of Child Health, London, UK

Abstract: The history of stem cell gene therapy is strongly linked to the development of gene therapy for severe combined immunodeficiencies (SCID) and especially adenosine deaminase (ADA)-deficient SCID. Here we discuss the developments achieved in over two decades of clinical and laboratory research that led to the establishment of a protocol for the autologous transplant of retroviral vector-mediated gene-modified hematopoietic stem cells, which has proved to be both successful and, to date, safe. Patients in trials in three different countries have shown long-term immunological and metabolic correction. Nevertheless, improvements to the safety profile of viral vectors are underway and will undoubtedly reinforce the position of stem cell gene therapy as a treatment option for ADA-SCID.

Keywords: adenosine deaminase, severe combined immunodeficiency, gene therapy, hematopoietic stem cell, retrovirus, clinical trial

Creative Commons License © 2009 The Author(s). This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License. By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.