Inventors:
Philippe LeBoulch - Cambridge MA
Irving M. London - Cambridge MA
Dorothy Tuan - Newton MA
Assignee:
Massachusetts Institute of Technology - Cambridge MA
International Classification:
C12N 1564, C12N 1568, C12N 1585, C12N 1586
Abstract:
A process and means for the design and the optimization of retroviral vectors transducing human. beta. -globin gene and. beta. -Locus Control Region (. beta. -LCR) derivatives, hereafter referred to as [. beta. -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. beta. -globin gene, are described, along with specific constructs.