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Liver cell transplantation for the treatment of inborn errors of metabolism
Author(s) -
Meyburg J.,
Hoffmann G. F.
Publication year - 2008
Publication title -
journal of inherited metabolic disease
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.462
H-Index - 102
eISSN - 1573-2665
pISSN - 0141-8955
DOI - 10.1007/s10545-008-0829-6
Subject(s) - human genetics , transplantation , liver transplantation , medicine , inborn error of metabolism , galactosemia , metabolic disease , cell metabolism , metabolism , bioinformatics , biology , genetics , biochemistry , galactose , gene
Summary 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 LCTin the treatment of metabolic diseases and call forcollaborative clinical research.