Gene therapy vectors are being used to effectively treat an increasing number of serious diseases. However, the relative low transduction efficiency in human long-term hematopoietic stem cells limits the utility of these vectors in this cell type. A new development may change this.
Researchers at St. Jude have discovered that overexpression of a truncated form of the human HMGA2 gene in a lentiviral vector can cause transduced hematopoietic stem cells to proliferate in transplanted animal models. By incorporating the HMGA2 gene, the small number of hematopoietic stem cells that were initially transduced (about 1%) expanded over a period of two years to become 40-70% of the stem cell population in transplant recipients. This approach can be used to achieve in vivo expansion of hematopoietic stem cells transduced with lentiviral vectors that carry any desired therapeutic gene, such as gamma-globin, or to enhance genome editing efficiency.
Gene therapy, lentiviral, hematopoietic stem cell, HMGA2
Granted Patents or Published Applications
International Publication WO 2017/156057
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