abstract |
The present invention relates to expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding anti-HPRT RNAi, and a nucleic acid sequence encoding a gamma globin gene. In some embodiments, the viral vector is a self-inactivating lentiviral vector. In some embodiments, the gamma globin gene is used to genetically correct sickle cell disease or β-thalassemia or reduce their symptoms. |