Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC
Mucopolysaccharidosis type IIIC (MPSIIIC) is a severe lysosomal storage disease caused by deficiency in activity of the transmembrane enzyme heparan-α-glucosaminide N-acetyltransferase (HGSNAT) that catalyses the N-acetylation of α-glucosamine residues of heparan sulfate. Enzyme deficiency causes ab...
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| Main Authors: | , , , , , , , , , , , , , , |
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| Format: | Book |
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The Company of Biologists,
2016-09-01T00:00:00Z.
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