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).
Preview |
PDF
1-s2.0-S2213219824000710-main.pdf - Published Version Download (704kB) | Preview |
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-pp.ucl.ac.uk/id/eprint/10188789 |
Archive Staff Only
 |
View Item |
