Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides.
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R. Kole | S. Fucharoen | H. Sierakowska | S. Kirby | S. Svasti | Thipparat Suwanmanee | G. Lacerra | C. Walsh