Lineage-specific expression of a human β-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells

• DOI: 10.1038/331035a0
• Publication Date: 2003-06-12T21:41:34Z
• Authors: Elaine A. Dzierzak; Thalia Papayannop...; Richard C. Mulligan
• Keywords: Male; 0301 basic medicine; Mice, Inbred C3H; Genetic Vectors; Hematopoietic Stem Cell Transplantation; Recombinant Proteins; Globins; 3. Good health; Mice; 03 medical and health sciences; Retroviridae; Gene Expression Regulation; Organ Specificity; Radiation Chimera; Animals; Humans; RNA, Viral; Female; RNA, Messenger; Cells, Cultured; Bone Marrow Transplantation

Recombinant retroviral genomes encoding a chromosomal human beta-globin gene have been used to transduce murine haematopoietic stem cells in vitro. After permanent engraftment of lethally irradiated recipients with the transduced cells, the human beta-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.