Table 1.

Patients Studied

Patient No. Mutations Age (yr) Duration of Therapy When AnalyzedNon-α:α Globin Chain SynthesisFold Increase in γ Globin mRNA on Therapy*
Baseline Treatment
1  β+ Thalassemia  4 wk  0.5  0.8  3.5  
 IVSI-6  
 codon 39  
β0 Thalassemia  41  2 wk  0.5  0.75 2.8  
 Frameshift 41/42  
 Frameshift 5  
β0 Thalassemia  27  1 wk  0.4  0.8 3.9  
 IVS I-1  
 IVS II-1  
4  β+Thalassemia  21  6 wk  0.4  0.5-151 1.0  
 codon 39/?  
5  β+ Thalassemia  6  3 wk  0.4 0.84  3.0  
 −87/codon 39  
6  β+Thalassemia  16  4 wk  0.4  0.9  6.1  
 IVS II 848 
7  β0 Thalassemia  25  6 wk  0.47 0.86  3.1  
 Homozygous  
 codon 39  
8  Hb SS 7  3 wk  0.5-152 11.5-152 5.2-152 
Patient No. Mutations Age (yr) Duration of Therapy When AnalyzedNon-α:α Globin Chain SynthesisFold Increase in γ Globin mRNA on Therapy*
Baseline Treatment
1  β+ Thalassemia  4 wk  0.5  0.8  3.5  
 IVSI-6  
 codon 39  
β0 Thalassemia  41  2 wk  0.5  0.75 2.8  
 Frameshift 41/42  
 Frameshift 5  
β0 Thalassemia  27  1 wk  0.4  0.8 3.9  
 IVS I-1  
 IVS II-1  
4  β+Thalassemia  21  6 wk  0.4  0.5-151 1.0  
 codon 39/?  
5  β+ Thalassemia  6  3 wk  0.4 0.84  3.0  
 −87/codon 39  
6  β+Thalassemia  16  4 wk  0.4  0.9  6.1  
 IVS II 848 
7  β0 Thalassemia  25  6 wk  0.47 0.86  3.1  
 Homozygous  
 codon 39  
8  Hb SS 7  3 wk  0.5-152 11.5-152 5.2-152 

*α-Globin mRNA was used as internal control in the β-thalassemia subjects.

F0-151

No changes were detected at the time that mRNA and nuclear extracts were analyzed.

F0-152

Represents γ/γ + β × 100 and non-α:α-globin ratios were balanced.

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