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- W2768977819 abstract "Hematopoietic stem cells possess the capacity of self-renewal and differentiation into all lineages of blood cells and have been recognized as one of the most ideal cells for gene therapy. Over the last decade, significant progress has been made in lentiviral vector-based hematopoietic stem cell gene therapy, and long-term success has been achieved in some clinical trials. The recent advent of the CRISPR-Cas9 genome editing technology has galvanized the field of gene therapy, and emerging on the horizon is targeted gene therapy by precise genome editing. However, to fulfill the vision of the second-generation hematopoietic stem cell gene therapy, further innovation is imperative to considerably increase the efficiency of precision genome editing in long-term hematopoietic stem cells." @default.
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- W2768977819 date "2017-12-01" @default.
- W2768977819 modified "2023-10-16" @default.
- W2768977819 title "Hematopoietic stem cell gene therapy: progress and challenges" @default.
- W2768977819 doi "https://doi.org/10.1360/n052017-00268" @default.
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