Sanfilippo Syndrome: Molecular Basis, Disease Models and Therapeutic Approaches
Sanfilippo syndrome or mucopolysaccharidosis III is a lysosomal storage disorder caused by mutations in genes responsible for the degradation of heparan sulfate, a glycosaminoglycan located in the extracellular membrane. Undegraded heparan sulfate molecules accumulate within lysosomes leading to cel...
Main Authors: | Noelia Benetó, Lluïsa Vilageliu, Daniel Grinberg, Isaac Canals |
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Format: | Article |
Language: | English |
Published: |
MDPI AG
2020-10-01
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Series: | International Journal of Molecular Sciences |
Subjects: | |
Online Access: | https://www.mdpi.com/1422-0067/21/21/7819 |
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