Ghosh, S;
Gaspar, HB;
(2017)
Gene Therapy Approaches to Immunodeficiency.
Hematology/Oncology Clinics of North America
, 31
(5)
pp. 823-834.
10.1016/j.hoc.2017.05.003.
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Abstract
Transfer of gene-corrected autologous haematopoietic stem cells in patients with primary immunodeficiencies has emerged as a new therapeutic approach in the last three decades. Patients with various conditions lacking a suitable donor for transplant have been treated with retroviral vectors and a gene-addition strategy. Initial promising results were shadowed by the occurrence of malignancies in some of these patients. Current trials, developed in the last decade, employ safer viral vectors to overcome the risk of genotoxicity and have led to improved clinical outcomes. This review reflects the progresses made in specific disorders including adenosine deaminase deficiency, X-linked severe combined immunodeficiency, chronic granulomatous disease and Wiskott-Aldrich syndrome. The success of these studies suggests that gene therapy has the potential to become a standard therapeutic option in the care of patients with these disorders.
| Type: | Article |
|---|---|
| Title: | Gene Therapy Approaches to Immunodeficiency |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1016/j.hoc.2017.05.003 |
| Publisher version: | https://doi.org/10.1016/j.hoc.2017.05.003 |
| Language: | English |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions. |
| Keywords: | Gene therapy; Primary immunodeficiency; Adenosine deaminase deficiency; X-linked severe combined immunodeficiency; Chronic granulomatous disease; Wiskott-Aldrich syndrome |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health |
| URI: | https://discovery.ucl.ac.uk/id/eprint/1564448 |
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