Hematopoietic Stem Cells Journals

Hematopoietic stem cells (HSCs) are the most routinely transplanted adult stem cell. Currently, they are utilized for the treatment of several genetic and acquired diseases including blood cancers, autoimmune disorders, and hematopoietic defects. Hematopoietic stem cells are ideal candidates for gene therapy applications because they possess the capacity for selfreplication and functionality to propagate the entire hematopoietic lineage, thus facilitating amplification of genetically-modified cells and expression of a transgene product from a multitude of hematopoietic cell types. An additional advantage is the tolerogenic effect Hematopoietic stem cells have on host immunity, which in many contexts, is a barrier to successful gene therapy. Numerous Hematopoietic stem cell-targeted gene therapy studies have been conducted in a range of disease settings. Current pre-clinical research for Hematopoietic stem cell transplantation gene therapy of hemophilia A therapy is focused on i) identification of safe and efficient methods of nucleic acid transfer into Hematopoietic stem cells, ii) optimization of the coagulation factor VIII transgene for high expression, iii) minimization of conditioning regimen-related toxicity with Hematopoietic stem cell engraftment and iv) overcoming complications due to pre-existing factor VIII immunity. Herein, we review the state of the art in Hematopoietic stem cell transplantation gene therapy of hemophilia A. (Zakas PM, Spencer HT, Doering CB, Engineered Hematopoietic Stem Cells as Therapeutics for Hemophilia A)

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Last date updated on April, 2025