Abstract
Lentiviral vectors for γ-globin genes are being developed as an efficient tool for the gene therapy of β-chain hemoglobinopathies. The γ-globin gene has been chosen as a therapeutic gene based on the potent anti-sickling properties of γ-globins and on their ability to bind free α-chains. However, their development has been hampered by low titers, variable expression and gene silencing. To address these problems, we have initiated a strategy to exploit novel regulatory elements of the β-cluster conferring high level and sustained globin gene expression. To this end, we have successfully used the HPFH-2 enhancer combined with a 210 bp Aγ-globin gene promoter harboring the Greek HPFH -117 mutation and the HS-40 enhancer from the α-globin locus, in a series of oncoretrovirus vectors (
Disclosure: No relevant conflicts of interest to declare.
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