Hematopoietic stem cell gene editing and expansion: State-of-the-art technologies and recent applications.
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Publication Date
2022-03Journal Title
Exp Hematol
ISSN
0301-472X
Publisher
Elsevier BV
Type
Article
This Version
AM
Physical Medium
Print-Electronic
Metadata
Show full item recordCitation
Haltalli, M., Wilkinson, A. C., Rodriguez-Fraticelli, A., & Porteus, M. (2022). Hematopoietic stem cell gene editing and expansion: State-of-the-art technologies and recent applications.. Exp Hematol https://doi.org/10.1016/j.exphem.2021.12.399
Abstract
Hematopoietic stem cell transplantation (HSCT) is a curative therapy for a range of hematological diseases, from leukemias to immunodeficiencies and anemias. The aim in using HSCT is to replace a patient's dysfunctional blood system with a functional one by transplanting healthy hematopoietic stem cells (HSCs). HSCs may be collected from a healthy donor (for allogeneic HSCT) or from the patient for genetic correction (for autologous HSCT gene therapies). Despite the curative potential of HSCT, several hurdles to its wider and safer use remain, including how to efficiently genetically correct HSCs and how to increase donor HSC numbers to improve the donor pool. In recent years, the development of state-of-the-art technologies, such as Cas9-AAV6 technologies and identification of the small molecule HSC agonist UM171, have accelerated progress in HSC gene editing and expansion. These translational research efforts were the focus of the Spring 2021 International Society for Experimental Hematology (ISEH) webinar. Here we present a summary and discussion of the implications of these new approaches to improve HSC-based therapy.
Sponsorship
Wellcome Trust (203151/Z/16/Z)
Medical Research Council (MC_PC_17230)
Embargo Lift Date
2022-12-29
Identifiers
External DOI: https://doi.org/10.1016/j.exphem.2021.12.399
This record's URL: https://www.repository.cam.ac.uk/handle/1810/332978
Rights
Attribution-NonCommercial-NoDerivatives 4.0 International
Licence URL: https://creativecommons.org/licenses/by-nc-nd/4.0/
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