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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...

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Detalles Bibliográficos
Autores principales:	Benetó, Noelia, Vilageliu, Lluïsa, Grinberg, Daniel, Canals, Isaac
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2020
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7659972/ https://www.ncbi.nlm.nih.gov/pubmed/33105639 http://dx.doi.org/10.3390/ijms21217819

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