Table 1.
Hepatocyte transplantation—clinical studies in liver-based metabolic diseases
| Disease | No. of patients | Effect/outcome | Reference |
|---|---|---|---|
| Familial hypercholesterolaemia | 5* | Some reduction in LDL in 3 patients | Grossman et al. (30) |
| Crigler–Najjar syndrome type I | 1 | 50% reduction in serum bilirubin; | Fox et al. (5) |
| 1 | 40% reduction in serum bilirubin | Dhawan et al. (unpublished) | |
| Urea cycle defect | 1 | Some clinical improvement; died after 42 days | Strom et al. (31) |
| 1 | Lowered blood ammonia and increased protein tolerance | Horslen et al. (7) | |
| 1 | No hyperammonaemia, increased urea synthesis | Mitry et al. (14) | |
| Infantile Refsum's disease | 1 | Partial correction of metabolic abnormality | Sokal et al. (32) |
| Glycogen storage disease type Ia | 1 | No hypoglycaemia on normal diet | Muraca et al. (6) |
| Factor VII deficiency | 2 | 80% reduction in recombinant factor VII requirement | Dhawan et al. (33) |
| PFIC2 | 2 | No clear benefit—fibrosis already present | Dhawan et al. (unpublished) |
LDL=low-density lipoprotein; PFIC2=progressive familial intrahepatic cholestasis
*
Ex-vivo gene therapy of autologous hepatocytes