The recombinant human parvoviruses for gene therapy of hemoglobinopathies.

Abstract

Towards a goal of using adeno-associated viruses (AAV), the human parvovirus, as the gene transfer vector for gene therapy of hemoglobinopathies, the human beta-globin (h beta G) cDNA was ligated downstream of the P40 promoter of AAV type 2 (AAV2) genome. Transfection via electroporation of the construct into human 293 cells (embryonal kidney cell line) resulted in expression of the cloned h beta G cDNA, as evidenced by the synthesis of transcripts hybridizable to h beta G probe. The transfection led to the recombinant genome to be excised out of the plasmid and replicate in the cell, followed by production of the recombinant AAV that harbors h beta G cDNA.