Table 1.
Patient ID | Age (years) | Gender | Phenotype | Reprogramming efficiency |
---|---|---|---|---|
WT1 | Newborn | Male | Heathy | 0.21% |
WT2 | 51 | Male | Healthy | 0.29% |
WT3 | 37 | Female | Healthy | 0.14% |
SAA1 | 16 | Male | Severe AA Responded to immunosuppressors | 0.11% |
SAA2 | 24 | Male | Very Severe AA Responded to immunosuppressors Developed PNH and relapsed Failed with second course of horse ATG Successful matched unrelated HSCT | 0.10% |
SAA3 | 10 | Female | Very Severe AA and autism Successful matched unrelated HSCT | 0.11% |
AA aplstic anemia, PNH paroxysmal nocturnal hemaoglobinuria, ATG anti-thymocyte globulin, HSCT hematopoietic stem cell transplantation