1. Hematopoietic stem cell gene therapy: The optimal use of lentivirus and gene editing approaches.
- Author
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Lamsfus-Calle A, Daniel-Moreno A, Ureña-Bailén G, Raju J, Antony JS, Handgretinger R, and Mezger M
- Subjects
- Autografts, Humans, Gene Editing, Genetic Therapy, Hematologic Diseases genetics, Hematologic Diseases therapy, Hematopoietic Stem Cell Transplantation, Hematopoietic Stem Cells, Immune System Diseases genetics, Immune System Diseases therapy, Lentivirus
- Abstract
Due to pioneering in vitro investigations on gene modification, gene engineering platforms have incredibly improved to a safer and more powerful tool for the treatment of multiple blood and immune disorders. Likewise, several clinical trials have been initiated combining autologous hematopoietic stem cell transplantation (auto-HSCT) with gene therapy (GT) tools. As several GT modalities such as lentivirus and gene editing tools have a long developmental path ahead to diminish its negative side effects, it is hard to decide which modality is optimal for treating a specific disease. Gene transfer by lentiviruses is the platform of choice for loss-of-mutation diseases, whereas gene correction/addition or gene disruption by gene editing tools, mainly CRISPR/Cas9, is likely to be more efficient in diseases where tight regulation is needed. Therefore, in this review, we compiled pertinent information about lentiviral gene transfer and CRISPR/Cas9 gene editing, their evolution to a safer platform for HSCT, and their applications on other types of gene disorders based on the etiology of the disease and cell fitness., Competing Interests: Declaration of Competing Interest None of the authors state any conflicts of interest., (Copyright © 2019 Elsevier Ltd. All rights reserved.)
- Published
- 2020
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