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Transplantation of gene-modified haematopoietic stem cells: Application and clinical considerations
Alessandrini, M; Krause, K. H; Speck, R. F; Pepper, M. S.
  • Alessandrini, M; s.af
  • Krause, K. H; s.af
  • Speck, R. F; s.af
  • Pepper, M. S; s.af
S. Afr. med. j. (Online) ; 109(8): 65-70, 2019. ilus
Article Dans Anglais | AIM | ID: biblio-1271232
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
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Indice: AIM (Afrique) Sujet Principal: République d'Afrique du Sud / Applications de l'informatique médicale / Cellules souches hématopoïétiques / Transplantation de cellules souches / Services de laboratoire d'analyses médicales / Système hématopoïétique Pays comme sujet: Afrique langue: Anglais Texte intégral: S. Afr. med. j. (Online) Année: 2019 Type: Article

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Recherche sur Google
Indice: AIM (Afrique) Sujet Principal: République d'Afrique du Sud / Applications de l'informatique médicale / Cellules souches hématopoïétiques / Transplantation de cellules souches / Services de laboratoire d'analyses médicales / Système hématopoïétique Pays comme sujet: Afrique langue: Anglais Texte intégral: S. Afr. med. j. (Online) Année: 2019 Type: Article