Gene Therapy For Beta-Thalassemia: Updated Perspectives | TACG – Dove Medical Press

Garyfalia Karponi,1,* Nikolaos Zogas2,*

1Department of Veterinary Medicine, Aristotle University of Thessaloniki, Thessaloniki, Greece; 2Department of Biology, Aristotle University of Thessaloniki, Thessaloniki, Greece

*These authors contributed equally to this work

Correspondence: Garyfalia KarponiLaboratory of Microbiology and Infectious Diseases, Faculty of Veterinary Medicine, Aristotle University of Thessaloniki, Thessaloniki 54124, GreeceTel +30 2310-999-956Fax +30 2310-999-934Email gkarponi@vet.auth.gr

Abstract: Allogeneic hematopoietic stem cell transplantation was until very recently, the only permanent curative option available for patients suffering from transfusion-dependent beta-thalassemia. Gene therapy, by autologous transplantation of genetically modified hematopoietic stem cells, currently represents a novel therapeutic promise, after many years of extensive preclinical research for the optimization of gene transfer protocols. Nowadays, clinical trials being held on a worldwide setting, have demonstrated that, by re-establishing effective hemoglobin production, patients may be rendered transfusion- and chelation-independent and evade the immunological complications that normally accompany allogeneic hematopoietic stem cell transplantation. The present review will offer a retrospective scope of the long way paved towards successful implementation of gene therapy for beta-thalassemia, and will pinpoint the latest strategies employed to increase globin expression that extend beyond the classic transgene addition perspective. A thorough search was performed using Pubmed in order to identify studies that provide a proof of principle on the aforementioned topic at a preclinical and clinical level. Inclusion criteria also regarded gene transfer technologies of the past two decades, as well as publications outlining the pitfalls that precluded earlier successful implementation of gene therapy for beta-thalassemia. Overall, after decades of research, that included both successes and pitfalls, the path towards a permanent, donor-irrespective cure for beta-thalassemia patients is steadily becoming a realistic approach.

Keywords: gene therapy, gene editing, thalassemia, mobilization, viral vectors, clinical trials, hematopoietic stem cells

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

See the article here:
Gene Therapy For Beta-Thalassemia: Updated Perspectives | TACG - Dove Medical Press

Related Post