Recombinant adeno-associated virus (rAAV)-mediated expression of a human gamma-globin gene in human progenitor-derived erythroid cells

Analysis of the efficacy of recombinant adeno-associated virus (rAAV) for gene therapy using a rAAV vector with human gamma-globin gene reveals that rAAV regulates human gamma-globin expression and is effective in expressing and transferring the globin gene in primary erythroid cells for gene therapy. The genetic transfer and expression result in a high level increase of hemoglobin factor (HbF) level. Reverse transcriptase PCR assay shows that the human erythroid burst-forming unit-derived colonies contain rAAV-transduced, mutationally marked Agamma-globin gene expression, confirming its role in the gene therapy of Hb disorders, thalassemia and sickle-cell anemia.