Title: Gene therapy: prospects for glycolipid storage diseases
Authors: Gieselmann, Volkmar ×
Matzner, Ulrich
Klein, Diana
Mansson, Jan Eric
D'Hooge, Rudi
DeDeyn, Peter D
Lüllmann Rauch, Renate
Hartmann, Dieter
Harzer, Klaus #
Issue Date: Jun-2003
Series Title: Philosophical transactions of the Royal Society of London. Series B, Biological sciences. vol:358 issue:1433 pages:921-925
Abstract: Lysosomal storage diseases comprise a group of about 40 disorders, which in most cases are due to the deficiency of a lysosomal enzyme. Since lysosomal enzymes are involved in the degradation of various compounds, the diseases can be further subdivided according to which pathway is affected. Thus, enzyme deficiencies in the degradation pathway of glycosaminoglycans cause mucopolysaccharidosis, and deficiencies affecting glycopeptides cause glycoproteinosis. In glycolipid storage diseases enzymes are deficient that are involved in the degradation of sphingolipids. Mouse models are available for most of these diseases, and some of these mouse models have been used to study the applicability of in vivo gene therapy. We review the rationale for gene therapy in lysosomal disorders and present data, in particular, about trials in an animal model of metachromatic leukodystrophy. The data of these trials are compared with those obtained with animal models of other lysosomal diseases.
ISSN: 0962-8436
Publication status: published
KU Leuven publication type: IT
Appears in Collections:Molecular Genetics Section (-)
Laboratory for Biological Psychology
× corresponding author
# (joint) last author

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