Current Topics in Human Hematopoietic Stem Cell Gene Therapy: Leukemogenesis:Leukemogenesis
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概要
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Over the past decade, clinical success in the treatment of serious genetic disorders using retroviral vectors was achieved, specifically in patients with serious genetic immunodeficiency diseases. However, this progress was unfortunately tempered by a serious adverse event, leukemogenesis, related to retroviral vector integration into genomes of hematopoietic stem cells or primitive precursor cells in 2003. Here, the encouraging results and the serious adverse event based on the genotoxicity of vector integration will be reviewed, and strategies to decrease the risk of leukemogenesis and adverse events to achieve clinical safe gene therapy using hematopoietic stem cells will be discussed.
- 特定非営利活動法人 日本小児血液・がん学会の論文
特定非営利活動法人 日本小児血液・がん学会 | 論文
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