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What a Clinician Needs to Know About Genome Editing: Status and Opportunities for Inborn Errors of Immunity

Mudde, Anne CA; Kuo, Caroline Y; Kohn, Donald B; Booth, Claire; (2024) What a Clinician Needs to Know About Genome Editing: Status and Opportunities for Inborn Errors of Immunity. The Journal of Allergy and Clinical Immunology: In Practice 10.1016/j.jaip.2024.01.019. (In press). Green open access

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Abstract

During the past 20 years, gene editing has emerged as a novel form of gene therapy. Since the publication of the first potentially therapeutic gene editing platform for genetic disorders, increasingly sophisticated editing technologies have been developed. As with viral vector-mediated gene addition, inborn errors of immunity are excellent candidate diseases for a corrective autologous hematopoietic stem cell gene editing strategy. Research on gene editing for inborn errors of immunity is still entirely preclinical, with no trials yet underway. However, with editing techniques maturing, scientists are investigating this novel form of gene therapy in context of an increasing number of inborn errors of immunity. Here, we present an overview of these studies and the recent progress moving these technologies closer to clinical benefit.

Type: Article
Title: What a Clinician Needs to Know About Genome Editing: Status and Opportunities for Inborn Errors of Immunity
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.jaip.2024.01.019
Publisher version: https://doi.org/10.1016/j.jaip.2024.01.019
Language: English
Additional information: © The Author(s), 2024. This is an Open Access article distributed under the terms of the Creative Commons Attribution Licence (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. https://creativecommons.org/licenses/by/4.0/
Keywords: Base editing, CRISPR/Cas, Gene editing, Inborn errors of immunity, Prime editing
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
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/10188789
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