Abstract
Recombinant retroviral genomes encoding a chromosomal human β-globin gene have been used to transduce murine haematopoietic stem cells in vitro. After permanent engraftment oflethally irradiated recipients with the transduced cells, the human β-globin gene is expressed at significant levels only within the erythroid lineage. These results indicate that it is possible to obtain stable expression of exogenous chromosomal DNA sequences introduced into mature haematopoietic cells in vivo via stem cell infection, and that human disorders of haemoglobin production may be more feasible candidates for somatic cell gene therapy than previously suspected.
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Dzierzak, E., Papayannopoulou, T. & Mulligan, R. Lineage-specific expression of a human β-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells . Nature 331, 35–41 (1988). https://doi.org/10.1038/331035a0
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DOI: https://doi.org/10.1038/331035a0
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