Incontinence is still a challenging problem in children with the exstrophy-epispadias complex (EEC) and satisfactory results using conventional management has not yet been achieved. Recent advances in cell therapy and tissue engineering techniques have been promising. We investigated autologous, heterotopic targeted muscle precursor cell donation in the recovery of external urethral sphincter function in children with the EEC and urinary incontinence (UI).Following extensive animal model studies at our laboratory and approval of the study protocol by the local bioethical committee, 5 children (4 boys and 1 girl, mean age: 9.6 years) with severe urinary incontinence due to exstrophy/epispadias were enrolled in May 2006. Autologous skeletal muscle precursor cells were obtained from the pelvic floor and cultured in vitro for cell expansion. Before transurethral injection of the cells into the sphincter, microbiological control and karyotyping were performed. The continence status of the patients prior to and following satellite injection was evaluated according to incontinence score, biochemical parameters, urodynamic studies and changes in quality of life.An average of 0.2-8x10 7 cells (>70% Pax7+/ MyoD+/ Desmin+) were obtained after a mean period of 18 days and implanted uneventfully. Neither microbial infection nor chromosomal change was detected in the specimens prior to cell implantation. During 4 months follow-up the incontinence score improved in 4 patients but 1 female patient with a very short urethral length and fibrosis had no change in continence status.This data confirms the potential application and outcome of satellite cell transplantation in functional reconstruction in UI by compensating for the cell loss. Although it can provide a new therapeutic approach for incontinence therapy with a reasonable success rate, long-term follow-up is still required.