Sanfilippo Syndrome: Molecular Basis, Disease Models and Therapeutic Approaches

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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Bibliographic Details
Main Authors: Noelia Benetó, Lluïsa Vilageliu, Daniel Grinberg, Isaac Canals
Format: Article
Language:English
Published: MDPI AG 2020-10-01
Series:International Journal of Molecular Sciences
Subjects:
Sanfilippo syndrome
mucopolysaccharidosis III
lysosomal storage disorders
heparan sulfate
animal models
induced pluripotent stem cells
Online Access:https://www.mdpi.com/1422-0067/21/21/7819

Internet

https://www.mdpi.com/1422-0067/21/21/7819

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