Retroviral vectors for transducing .beta.-globin gene and .beta.

Chemistry: molecular biology and microbiology – Vector – per se

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435 691, 4351723, C12N 1564, C12N 1568, C12N 1585, C12N 1586

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056311621

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.

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