Gene therapy for rat renal anemia with implantation of erythropoietin-transgenic myoblasts

To investigate whether an erythropoietin (EPO) gene-based therapy could ser ve as an alternative to the repeated injection of rhEPO in treatment to ren al anemia, the genetically modified myoblasts oi rats, named Myo/EPO, were implanted through intramuscular injection to model rats with renal anemia. The hemoglobin (Hb) and hematocrit (HCT) of the rats increased from (92.5 /- 3.0) g/L and 0.29+/-0.04 to the peak values oi (103.8 +/-5.0) g/L and 0. 32 +/- 0.04 respectively 14 d after implantation, and sustained the pre-imp lantation level for 90 d. Otherwise, the control rats implanted with Myo/X, which carried the parent retroviral vector, gradually became severe in ane mia. The PCR detection for hEPO cDNA in the rat muscle adjacent to injectio n sites indicated that the Myo/EPO cells survived for a long period in the muscle of rats. The results primarily demonstrate that myoblast gene transf er of EPO is effective for the treatment of rat renal anemia.