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dc.contributor.authorHaltalli, Myriam LR
dc.contributor.authorWilkinson, Adam C
dc.contributor.authorRodriguez-Fraticelli, Alejo
dc.contributor.authorPorteus, Matthew
dc.date.accessioned2022-01-28T00:31:08Z
dc.date.available2022-01-28T00:31:08Z
dc.date.issued2022-03
dc.identifier.issn0301-472X
dc.identifier.urihttps://www.repository.cam.ac.uk/handle/1810/332978
dc.description.abstractHematopoietic 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.
dc.format.mediumPrint-Electronic
dc.publisherElsevier BV
dc.rightsAttribution-NonCommercial-NoDerivatives 4.0 International
dc.rights.urihttps://creativecommons.org/licenses/by-nc-nd/4.0/
dc.titleHematopoietic stem cell gene editing and expansion: State-of-the-art technologies and recent applications.
dc.typeArticle
dc.publisher.departmentDepartment of Haematology
dc.date.updated2022-01-27T13:35:55Z
prism.publicationDate2021
prism.publicationNameExp Hematol
dc.identifier.doi10.17863/CAM.80402
dcterms.dateAccepted2021-12-23
rioxxterms.versionofrecord10.1016/j.exphem.2021.12.399
rioxxterms.versionAM
dc.contributor.orcidHaltalli, Myriam [0000-0002-0886-4466]
dc.identifier.eissn1873-2399
rioxxterms.typeJournal Article/Review
pubs.funder-project-idWellcome Trust (203151/Z/16/Z)
pubs.funder-project-idMedical Research Council (MC_PC_17230)
cam.issuedOnline2021-12-29
cam.orpheus.success2022-01-27 - Embargo set during processing via Fast-track
cam.depositDate2022-01-27
pubs.licence-identifierapollo-deposit-licence-2-1
pubs.licence-display-nameApollo Repository Deposit Licence Agreement
rioxxterms.freetoread.startdate2022-12-29


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Attribution-NonCommercial-NoDerivatives 4.0 International
Except where otherwise noted, this item's licence is described as Attribution-NonCommercial-NoDerivatives 4.0 International