Selective neurodegeneration in murine mucopolysaccharidosis VII is progressive and reversible

The mucopolysaccharidoses are caused by inherited deficiencies of lysosomal enzymes involved in the degradative pathway of glycosaminoglycans. Lysosomal storage leads to cellular and organ dysfunction, including mental retardation. Storage lesions are found throughout the diseased brain, but little...

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Veröffentlicht in:	Annals of neurology 2002-12, Vol.52 (6), p.762-770
Hauptverfasser:	Heuer, Gregory G., Passini, Marco A., Jiang, Kanli, Parente, Michael K., Lee, Virginia M.-Y., Trojanowski, John Q., Wolfe, John H.
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Biological and medical sciences Brain - drug effects Brain - pathology Carbohydrates (enzymatic deficiencies). Glycogenosis Disease Progression Errors of metabolism Genetic Vectors - pharmacology Genetic Vectors - therapeutic use Inclusion Bodies - drug effects Inclusion Bodies - pathology Medical sciences Metabolic diseases Mice Mice, Inbred C3H Mice, Mutant Strains Mucopolysaccharidosis VII - drug therapy Mucopolysaccharidosis VII - genetics Mucopolysaccharidosis VII - pathology Neurodegenerative Diseases - drug therapy Neurodegenerative Diseases - genetics Neurodegenerative Diseases - pathology Ubiquitin - analysis Vaccines, Synthetic - pharmacology Vaccines, Synthetic - therapeutic use
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