Gene Therapy: Prospects for Glycolipid Storage Diseases

Overview

Journal Philos Trans R Soc Lond B Biol Sci

Specialty Biology

Date 2003 Jun 14

PMID 12803926

Citations 7

Authors

Volkmar Gieselmann

Ulrich Matzner

Diana Klein

Jan Eric Mansson

Rudi DHooge

Peter D DeDeyn

Renate Lullmann Rauch

Dieter Hartmann

Klaus Harzer

Affiliations

Soon will be listed here.

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.

Citing Articles

Gene therapy for lysosomal storage diseases (LSDs) in large animal models.

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The role and metabolism of sulfatide in the nervous system.

Eckhardt M Mol Neurobiol. 2008; 37(2-3):93-103.

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A historical perspective of the glycosphingolipids and sphingolipidoses.

Watts R Philos Trans R Soc Lond B Biol Sci. 2003; 358(1433):975-83.

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Future perspectives for glycolipid research in medicine.

Cox T Philos Trans R Soc Lond B Biol Sci. 2003; 358(1433):967-73.

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Substrate reduction therapy in mouse models of the glycosphingolipidoses.

Platt F, Jeyakumar M, Andersson U, Heare T, Dwek R, Butters T Philos Trans R Soc Lond B Biol Sci. 2003; 358(1433):947-54.

PMID: 12803928 PMC: 1693185. DOI: 10.1098/rstb.2003.1279.

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