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Genome editing for primary immunodeficiencies: A therapeutic perspective on Wiskott-Aldrich syndrome

Naseem, Asma; Steinberg, Zohar; Cavazza, Alessia; (2022) Genome editing for primary immunodeficiencies: A therapeutic perspective on Wiskott-Aldrich syndrome. Frontiers in Immunology , 13 , Article 966084. 10.3389/fimmu.2022.966084. Green open access

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Abstract

Primary immunodeficiency diseases (PIDs) are a group of rare inherited disorders affecting the immune system that can be conventionally treated with allogeneic hematopoietic stem cell transplantation and with experimental autologous gene therapy. With both approaches still facing important challenges, gene editing has recently emerged as a potential valuable alternative for the treatment of genetic disorders and within a relatively short period from its initial development, has already entered some landmark clinical trials aimed at tackling several life-threatening diseases. In this review, we discuss the progress made towards the development of gene editing-based therapeutic strategies for PIDs with a special focus on Wiskott - Aldrich syndrome and outline their main challenges as well as future directions with respect to already established treatments.

Type: Article
Title: Genome editing for primary immunodeficiencies: A therapeutic perspective on Wiskott-Aldrich syndrome
Location: Switzerland
Open access status: An open access version is available from UCL Discovery
DOI: 10.3389/fimmu.2022.966084
Publisher version: https://doi.org/10.3389/fimmu.2022.966084
Language: English
Additional information: © 2022 Naseem, Steinberg and Cavazza. This is an open-access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/).
Keywords: CRISPR/Cas9, Wiskott-Aldrich syndrome, gene therapy, genome editing, primary immunodeficiency, rare diseases
UCL classification: UCL
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
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
URI: https://discovery.ucl.ac.uk/id/eprint/10155536
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