Genetically Modulated Substrate Reduction Therapy for Sanfilippo syndrome – proof of principle

Introduction: Mucopolysaccharidosis type III (MPS III) refers to a group of five autosomal recessive neurodegenerative lysosomal storage disorders caused by the incomplete lysosomal degradation of the heparan sulphate (HS) that accumulates in patient cells and triggers disease. The main characterist...

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Bibliographic Details
Main Author: Santos, J.I. (author)
Other Authors: Coutinho, Maria Francisca (author), Gaspar, P. (author), Alves, S. (author)
Format: conferenceObject
Language:eng
Published: 2021
Subjects:
Lysosomal Storage Diseases
Mucopilysaccharidoses
Substrate Reduction Therapy
RNAi
Doenças Genéticas
Genética Humana
Online Access:http://hdl.handle.net/10400.18/7454
Country:Portugal
Oai:oai:repositorio.insa.pt:10400.18/7454

Internet

http://hdl.handle.net/10400.18/7454

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