Transplantation of gene-modified haematopoietic stem cells: Application and clinical considerations

Alessandrini, M; Krause, K-H; Speck, R.F; Pepper, M.S

Publication:
Transplantation of gene-modified haematopoietic stem cells: Application and clinical considerations

dc.contributor.author	Alessandrini, M
dc.contributor.author	Krause, K-H
dc.contributor.author	Speck, R.F
dc.contributor.author	Pepper, M.S
dc.contributor.department	M S Pepper, SAMRC Extramural Unit for Stem Cell Research and Therapy, Institute for Cellular and Molecular Medicine, Department of Immunology, Faculty of Health Sciences, University of Pretoria, South Africa	en_US
dc.date.accessioned	2024-06-09T15:02:37Z
dc.date.available	2024-06-09T15:02:37Z
dc.date.epub	2019
dc.date.issued	2019-09-10
dc.description.abstract	Autologous and allogeneic haematopoietic stem cell (HSC) transplantation has been performed in patients with various malignant and non-malignant haematological disorders for more than 50 years. Ex vivo gene modification of HSCs for autologous transplantation opens up new therapeutic avenues for genetic and infectious diseases. Major advances have been made over the last three decades with respect to gene modification of HSCs and transplantation strategies, ultimately culminating in the approval of two such therapies in Europe (Strimvelis for a rare primary immune deficiency, and LentiGlobin for beta-thalassaemia). Newer gene-modifying technologies and treatment regimens have also recently come to the fore, which hold great promise for the development of safer and more effective treatments. We provide an overview of the current state of gene-modified HSC therapies, highlighting success stories, limitations and important considerations for achieving successful translation of these therapies to the clinic.	en_US
dc.identifier.citation	Alessandrini M, Krause KH, Speck RF, Pepper MS. Transplantation of gene-modified haematopoietic stem cells: Application and clinical considerations. S Afr Med J. 2019 Sep 10;109(8b):64-69. doi: 10.7196/SAMJ.2019.v109i8b.013910.	en_US
dc.identifier.doi	10.7196/SAMJ.2019.v109i8b.013910
dc.identifier.journal	South African Medical Journal	en_US
dc.identifier.other	10.7196/SAMJ.2019.v109i8b.013910
dc.identifier.uri	http://www.samj.org.za/index.php/samj/article/view/12717
dc.identifier.uri	https://hdl.handle.net/11288/596469
dc.language.iso	en	en_US
dc.publisher	South African Medical Journal (SAMJ)
dc.research.unit	Stem Cell Research and Therapy	en_US
dc.rights	Attribution 3.0 United States	*
dc.rights.uri	http://creativecommons.org/licenses/by/3.0/us/	*
dc.subject	Gene-modified haematopoietic stem cells
dc.subject	Gene-modifying technologies
dc.subject	Haematological disorders
dc.title	Transplantation of gene-modified haematopoietic stem cells: Application and clinical considerations	en_US
dc.type	Article	en_US
dspace.entity.type	Publication