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Long-term expression of γ-globin mRNA in mouse erythrocytes from retrovirus vectors containing the human γ-globin gene fused to the ankyrin-1 promoter

Gene therapy for patients with hemoglobin disorders has been hampered by the inability of retrovirus vectors to transfer globin genes and their cis-acting regulatory sequences into hematopoietic stem cells without rearrangement. In addition, the expression from intact globin gene vectors has been va...

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Udgivet i:Proc Natl Acad Sci U S A
Principais autores: Sabatino, Denise E., Seidel, Nancy E., Aviles-Mendoza, Guillermo J., Cline, Amanda P., Anderson, Stacie M., Gallagher, Patrick G., Bodine, David M.
Format: Artigo
Sprog:Inglês
Udgivet: National Academy of Sciences 2000
Fag:
Biological Sciences
Online adgang:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC27218/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/11069298/
https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.230453097
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