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Gene Therapy in Cellular Immunodeficiencies

Fox, T; Booth, C; Morris, E; (2021) Gene Therapy in Cellular Immunodeficiencies. In: Cellular Primary Immunodeficiencies. (pp. 473-497). Springer Green open access

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Abstract

The treatment of cellular primary immunodeficiencies has benefitted from significant advances in the field of allogeneic stem cell transplantation (alloHSCT). However, while this therapy is curative for many PIDs, the procedure requires a suitably matched donor and carries significant risks of morbidity and mortality from complications such as graft-versus-host disease (GVHD). Autologous gene therapy (GT) approaches using stem cells isolated from patients and modified ex vivo using viral vectors or gene editing techniques have the potential to offer curative therapy for PID without the immunological complications of alloHSCT. GT for PID has been developed over the last 30 years, and while several setbacks have been encountered along the way, there is now a licensed GT product for ADA-SCID, and promising results from phase I/II clinical trials have demonstrated that GT may offer clinical efficacy comparable to alloHSCT in several other PIDs. Developments in the field are broadening the application of GT, and we expect that this therapeutic modality may become standard of care for the management of several PIDs in the near future. This chapter explores the development of GT over the last 30 years and outlines its role in the management of cellular primary immunodeficiencies.

Type: Book chapter
Title: Gene Therapy in Cellular Immunodeficiencies
ISBN-13: 978-3-030-70106-2
Open access status: An open access version is available from UCL Discovery
DOI: 10.1007/978-3-030-70107-9_19
Publisher version: https://doi.org/10.1007/978-3-030-70107-9_19
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.
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 Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Infection and Immunity
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10129934
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