Towards β-globin gene-targeting with integrase-defective lentiviral vectors
2010
Inanlou, Davoud Nouri | Yakhchali, Bagher | Khanahmad, Hossein | Gardaneh, Mossa | Movassagh, Hesam | Cohan, Reza Ahangari | Ardestani, Mehdi Shafiee | Mahdian, Reza | Zeinali, Sirous
We have developed an integrase-defective lentiviral (LV) vector in combination with a gene-targeting approach for gene therapy of β-thalassemia. The β-globin gene-targeting construct has two homologous stems including sequence upstream and downstream of the β-globin gene, a β-globin gene positioned between hygromycin and neomycin resistant genes and a herpes simplex virus type 1 thymidine kinase (HSVtk) suicide gene. Utilization of integrase-defective LV as a vector for the β-globin gene increased the number of selected clones relative to non-viral methods. This method represents an important step toward the ultimate goal of a clinical gene therapy for β-thalassemia.
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