Megakaryocyte- and megakaryocyte precursor-related gene therapies.

Blood
David A Wilcox

Abstract

Hematopoietic stem cells (HSCs) can be safely collected from the body, genetically modified, and re-infused into a patient with the goal to express the transgene product for an individual's lifetime. Hematologic defects that can be corrected with an allogeneic bone marrow transplant can theoretically also be treated with gene replacement therapy. Because some genetic disorders affect distinct cell lineages, researchers are utilizing HSC gene transfer techniques using lineage-specific endogenous gene promoters to confine transgene expression to individual cell types (eg, ITGA2B for inherited platelet defects). HSCs appear to be an ideal target for platelet gene therapy because they can differentiate into megakaryocytes which are capable of forming several thousand anucleate platelets that circulate within blood vessels to establish hemostasis by repairing vascular injury. Platelets play an essential role in other biological processes (immune response, angiogenesis) as well as diseased states (atherosclerosis, cancer, thrombosis). Thus, recent advances in genetic manipulation of megakaryocytes could lead to new and improved therapies for treating a variety of disorders. In summary, genetic manipulation of megakaryocytes has progr...Continue Reading

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Citations

Nov 14, 2017·Human Gene Therapy. Clinical Development·Terence R FlotteCheryl L McDonald
Dec 9, 2016·Journal of Thrombosis and Haemostasis : JTH·L J Latorre-ReyU Modlich
Jan 27, 2019·Scientific Reports·S NovakowskiC Kastrup
Jan 14, 2021·Cellular and Molecular Life Sciences : CMLS·Naoshi Sugimoto, Koji Eto
May 1, 2021·International Journal of Molecular Sciences·Verónica Palma-BarquerosJosé Rivera

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