Retroviral vectors for transducing amp;bgr;-globin gene and amp;bgr;- locus control region derivatives

摘要

A process and means for the design and the optimization of retroviral vectors transducing human &bgr;-globin gene and &bgr;-Locus Control Region (&bgr;-LCR) derivatives, hereafter referred to as [&bgr;- globin/LCR] retroviruses, which successfully meet the following criteria required for gene therapy applications: (1) stability of proviral transmission (low frequency of rearrangements similar to retroviral vectors considered stable in the art) upon infection of cell-lines and murine bone marrow cells, (2) improved viral titer, thereby allowing successful infection of bone marrow cells, and (3) high erythroid expression of the transduced human &bgr;-globin gene, are described, along with specific constructs.