ReviewInternational journal of molecular sciences2026
Oncogenesis as an Adverse Effect of Gene Replacement Therapy in Hematopoietic Stem Cells.
Review in International journal of molecular sciences, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Authors and funding
2 authors.
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Abstract
Genetically modified hematopoietic stem cell therapy using gene-modified autologous hematopoietic stem cells has evolved over the last 30 years as an alternative approach to circumvent the limitations of donor availability, risks of excessive regimen related toxicity, prolonged immune suppression and graft-versus-host disease associated with allogeneic hematopoietic cell transplantation. Gene replacement therapy based on viral insertion of transgene into host genome was developed as one of the main methods for gene modification of autologous cells. Unfortunately, many cases of oncogenesis were directly caused by genetically modified hematopoietic stem cell therapy. The purpose of the present review is the description of cases of leukemogenesis in gene replacement therapy in hematopoietic stem cells, elucidation of the causes, and overview of the risk mitigation strategies. It aims to elucidate the main risk factors in gene replacement therapy in hematopoietic stem cells. The insertional mutagenesis leads to activation of proto-oncogenes, mostly
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