Patients Studied
Patient No. . | Mutations . | Age (yr) . | Duration of Therapy When Analyzed . | Non-α:α Globin Chain Synthesis . | Fold Increase in γ Globin mRNA on Therapy* . | |
---|---|---|---|---|---|---|
Baseline . | Treatment . | |||||
1 | β+ Thalassemia | 3 | 4 wk | 0.5 | 0.8 | 3.5 |
IVSI-6 | ||||||
codon 39 | ||||||
2 | β0 Thalassemia | 41 | 2 wk | 0.5 | 0.75 | 2.8 |
Frameshift 41/42 | ||||||
Frameshift 5 | ||||||
3 | 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 Analyzed . | Non-α:α Globin Chain Synthesis . | Fold Increase in γ Globin mRNA on Therapy* . | |
---|---|---|---|---|---|---|
Baseline . | Treatment . | |||||
1 | β+ Thalassemia | 3 | 4 wk | 0.5 | 0.8 | 3.5 |
IVSI-6 | ||||||
codon 39 | ||||||
2 | β0 Thalassemia | 41 | 2 wk | 0.5 | 0.75 | 2.8 |
Frameshift 41/42 | ||||||
Frameshift 5 | ||||||
3 | 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 |