Arylsulfatase A knock-out mice

Ref.-Nr. 3206 - 4908

These mice can be used as a model to study metachromatic leukodystrophy, which is caused by a loss of arylsulfatase A function and consequently by sulfatide accumulation.

The sulfatide storage in arylsulfatase A-deficient mice is comparable to humans, but the mice do not mimic the myelin pathology. Therefore, in addition to the sole ASA-deficient knock-out mice (4908), a second, transgenic model was generated further overexpressing the sulfatide-synthesizing enzyme galactose-3-O-sulfotransferase (3206). These mice displayed a significant increase in sulfatide storage in the brain and peripheral nerves.

Genetic Background
Knock-out of the gene enocding for arylsulfatase A (4908), additionally overexpressing the sulfatide-synthesizing enzyme galactose-3-O-sulfotransferase by knock-in of the according gene Gal3st1 (3206).

Research, drug development

Hess, B., et al. (1996) Phenotype of arylsulfatase A-deficient mice: Relationship to human metachromatic leukodystrophy. Proc. Natl. Acad. Sci. USA 93: 14821-6.

Ramakrishnan, H., et al. (2007) Increasing sulfatide synthesis in myelin-forming cells of arylsulfatase A-deficient mice causes demyelination and neurological symptoms reminiscent of human metachromatic leukodystrophy, J. Neurosci, 27(35): 9482-90.

Prof. Dr. Frank Entschladen
+49 208 9410520