Figure 5.

Correction of splicing of β-globin pre-mRNA in mononuclear cells from IVS2–745 homozygote (DD) and IVS2–745/IVS2–1 heterozygote (ZA) thalassemic patients. On days 7 (A) and 14 (B) of the of culture, the cells from patient DD were treated for 24 h with morpholino oligomers indicated above the lanes. N, RNA from normal blood (lanes 9 and 5 in A and B, respectively). Oligonucleotide targeted to the IVS2–654 aberrant 5′-splice site, uninvolved in splicing of IVS2–745 pre-mRNA served as negative control (lane 2 in A and B). (C and D) Analogous treatments of 7- and 14-day cell cultures, respectively, from patient ZA. α, Oligonucleotide targeted to human α-globin gene as negative control (D, lane 9).