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

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

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

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

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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

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