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 |