Liver cell transplantation for the treatment of inborn errors of metabolism.
Over the last 15 years, liver cell transplantation (LCT) has developed from an experimental laboratory technique to a potentially life-saving therapeutic option. Because of its minimally invasive nature, the method is especially attractive for (small) children. In children with liver-based inborn errors of metabolism, this transfer of enzyme activity can be regarded as a gene therapy, which can be installed independently and additionally to conservative treatment concepts. To date 14 children with inherited metabolic diseases have undergone LCT in various centres. Although individual results are encouraging, different treatment protocols, difficulties in the objective assessment of function of the transplant, and finally the lack of a controlled study make it difficult to judge the overall significance of LCT in the treatment of metabolic diseases and call for collaborative clinical research.
Meyburg
J
J
Department of General Pediatrics, University Children’s Hospital, Heidelberg, Germany. jochen.meyburg@med.uni-heidelberg.de
Hoffmann
G F
GF
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods*; D006084:Graft Rejection / Q000209:etiology; D006085:Graft Survival; D022781:Hepatocytes / Q000637:transplantation*; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008099:Liver / Q000201:enzymology / Q000601:surgery*; D016031:Liver Transplantation / Q000009:adverse effects / Q000379:methods*; D008661:Metabolism, Inborn Errors / Q000201:enzymology / Q000235:genetics / Q000601:surgery*; D008875:Middle Aged; D016896:Treatment Outcome