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Gene therapy for primary immunodeficiencies

Fox, TA; Booth, C; (2021) Gene therapy for primary immunodeficiencies. British Journal of Haematology , 193 (6) pp. 1044-1059. 10.1111/bjh.17269. Green open access

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Abstract

Primary immunodeficiencies (PIDs) are a group of rare inherited disorders of the immune system. Many PIDs are devastating and require a definitive therapy to prevent progressive morbidity and premature mortality. Allogeneic haematopoietic stem cell transplantation (alloHSCT) is curative for many PIDs, and while advances have resulted in improved outcomes, the procedure still carries a risk of mortality and morbidity from graft failure or graft‐versus‐host disease (GvHD). Autologous haematopoietic stem cell gene therapy (HSC GT) has the potential to correct genetic defects across haematopoietic lineages without the complications of an allogeneic approach. HSC GT for PID has been in development for the last two decades and the first licensed HSC‐GT product for adenosine deaminase‐deficient severe combined immunodeficiency (ADA‐SCID) is now available. New gene editing technologies have the potential to circumvent some of the problems associated with viral gene‐addition. HSC GT for PID shows great promise, but requires a unique approach for each disease and carries risks, notably insertional mutagenesis from gamma‐retroviral gene addition approaches and possible off‐target toxicities from gene‐editing techniques. In this review, we discuss the development of HSC GT for PID and outline the current state of clinical development before discussing future developments in the field.

Type: Article
Title: Gene therapy for primary immunodeficiencies
Open access status: An open access version is available from UCL Discovery
DOI: 10.1111/bjh.17269
Publisher version: https://doi.org/10.1111/bjh.17269
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 immunodeficiencies, stem cell transplantation
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/10120113
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