dc.contributor.author | Gorur, V | |
dc.contributor.author | Kranc, KR | |
dc.contributor.author | Ganuza, M | |
dc.contributor.author | Telfer, P | |
dc.date.accessioned | 2024-07-04T07:06:04Z | |
dc.date.available | 2023-10-04 | |
dc.date.available | 2024-07-04T07:06:04Z | |
dc.date.issued | 2024-01-20 | |
dc.identifier.citation | Vishaka Gorur, Kamil R. Kranc, Miguel Ganuza, Paul Telfer, Haematopoietic stem cell health in sickle cell disease and its implications for stem cell therapies and secondary haematological disorders, Blood Reviews, Volume 63, 2024, 101137, ISSN 0268-960X, https://doi.org/10.1016/j.blre.2023.101137. (https://www.sciencedirect.com/science/article/pii/S0268960X2300098X) Abstract: Gene modification of haematopoietic stem cells (HSCs) is a potentially curative approach to sickle cell disease (SCD) and offers hope for patients who are not eligible for allogeneic HSC transplantation. Current approaches require in vitro manipulation of healthy autologous HSC prior to their transplantation. However, the health and integrity of HSCs may be compromised by a variety of disease processes in SCD, and challenges have emerged in the clinical trials of gene therapy. There is also concern about increased susceptibility to haematological malignancies during long-term follow up of patients, and this raises questions about genomic stability in the stem cell compartment. In this review, we evaluate the evidence for HSC deficits in SCD and then discuss their potential causation. Finally, we suggest several questions which need to be addressed in order to progress with successful HSC manipulation for gene therapy in SCD. Keywords: Sickle cell disease; Haematopoietic stem cell; Haematopoietic stem cell health; Stem cell therapies; Haematological malignancy in sickle cell disease | en_US |
dc.identifier.uri | https://qmro.qmul.ac.uk/xmlui/handle/123456789/97819 | |
dc.description.abstract | Gene modification of haematopoietic stem cells (HSCs) is a potentially curative approach to sickle cell disease (SCD) and offers hope for patients who are not eligible for allogeneic HSC transplantation. Current approaches require in vitro manipulation of healthy autologous HSC prior to their transplantation. However, the health and integrity of HSCs may be compromised by a variety of disease processes in SCD, and challenges have emerged in the clinical trials of gene therapy. There is also concern about increased susceptibility to haematological malignancies during long-term follow up of patients, and this raises questions about genomic stability in the stem cell compartment. In this review, we evaluate the evidence for HSC deficits in SCD and then discuss their potential causation. Finally, we suggest several questions which need to be addressed in order to progress with successful HSC manipulation for gene therapy in SCD. | en_US |
dc.format.extent | 101137 - ? | |
dc.language | eng | |
dc.publisher | Elsevier | en_US |
dc.relation.ispartof | Blood Rev | |
dc.rights | This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/). | |
dc.subject | Haematological malignancy in sickle cell disease | en_US |
dc.subject | Haematopoietic stem cell | en_US |
dc.subject | Haematopoietic stem cell health | en_US |
dc.subject | Sickle cell disease | en_US |
dc.subject | Stem cell therapies | en_US |
dc.subject | Humans | en_US |
dc.subject | Anemia, Sickle Cell | en_US |
dc.subject | Hematopoietic Stem Cells | en_US |
dc.subject | Hematologic Diseases | en_US |
dc.subject | Stem Cell Transplantation | en_US |
dc.subject | Hematopoietic Stem Cell Transplantation | en_US |
dc.title | Haematopoietic stem cell health in sickle cell disease and its implications for stem cell therapies and secondary haematological disorders. | en_US |
dc.type | Article | en_US |
dc.rights.holder | Crown Copyright © 2023 Published by Elsevier Ltd. | |
dc.identifier.doi | 10.1016/j.blre.2023.101137 | |
pubs.author-url | https://www.ncbi.nlm.nih.gov/pubmed/37919142 | en_US |
pubs.notes | Not known | en_US |
pubs.publication-status | Published | en_US |
pubs.volume | 63 | en_US |
dcterms.dateAccepted | 2023-10-04 | |
rioxxterms.funder | Default funder | en_US |
rioxxterms.identifier.project | Default project | en_US |
qmul.funder | MRC Career Development Award::Medical Research Council | en_US |