Disease Patient Age Number of viable cells transplanted Type of cell transplanted Outcome Study/Ref 8 years 7.5x109 F/C 40% decrease bilirubin up to 6 months; OLT at 20 months [15 ] 9 years 7.5x109 F 32% decrease in bilirubin for a few weeks; OLT after 5 months [16 ] 18 months 4.3x109 C 40% decrease in bilirubin to 7 months; OLT at 8 months [17 ] 8 years 1.4x109 F 30% decrease in bilirubin; OLT after 11 months [18 ] 1 year 2.6x109 F/C 25% decrease in bilirubin at 4 months; OLT after 4 months [19 ] 9 years 6.1x109 F/C 35% decrease in bilirubin at 6 months; OLT waiting list Glycogen storage disease type I (GSD) 18 years 6.0x109 F/C Improvement in glucose control, normal enzyme levels on biopsy [20 ] 47 years 2.0x109 F Better fasting time, decrease in triglycerides up to 18 months [21 ] Infantile Refsum’s disease 4 years 2.0x109 F/C 40% decrease pipecholic acid after 18 months [22 ] Familial hyper-cholesterolemia Five patients between 7 and 41 years 1.1x109 F 20% decrease in cholesterol, LDL, ApoB to 28 month; [23 ] 1.3x109 No effect; 1.0x109 6% decrease in cholesterol, LDL, ApoB to 19 months; 3.2x109 Minor effect 1.5x109 20% decrease in cholesterol up to 7 months Urea cycle defects OTC 5 years 1.0x109 F Decreased ammonia initially; Died 42 days later [24 ] OTC 10 hours 9.0x109 F/C Decrease ammonia; increased protein tolerance for a short period; OLT at 6 months [25 ] OTC 2 dias 1.9x109 F/C Decreased ammonia; auxiliary transplant at 6 months [26 ] OTC 14 months 2.4x109 C Decreased ammonia; increased urea; psychomotor improvement; OLT after 6 months [27 ] OTC 1 day 1.6x109 F/C Decreased ammonia; increased urea under normal diet; partial orthotopic liver transplantation (apolt) at 7 months [28 ] OTC 10 weeks 3 years 3.0x109 F Some stabilization [29 ] OTC 6 hours 0.6x1 C Decreased ammonia; increased urea; normal urinary orotic acid excretion. Death at 4 months [30 ] 9 days Decreased ammonia; increased protein intake, urinary orotic acid normal at 6 months. OLT waiting list Citrullinemia 36 months 1.5x1 C Normal ammonia; 40% increase in urea [30 ] Factor VII deficiency 3 months 35 months 1.1x109 C 70% decrease in recombinant factor VII for 6 months; OLT after 7 months [31 ] 2.2X109 F/C Phenylketonuria 6 years Two infusions for a total of 630 × 106 cells F blood phenylalanine levels returned within the therapeutic target while the phenylalanine half-life assessed by loading tests decreased from 43 to 19 h. [32 ]