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

Introduction: Sanfilippo syndrome, or Mucopolysaccharidosis (MPS) type III refers to a group of five autosomal recessive neurodegenerative lysosomal storage disorders caused by the incomplete lysosomal degradation of the glycosaminoglycan (GAG) heparan sulphate (HS) that accumulates in patient cells...

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Bibliographic Details
Main Author: Santos, Juliana Inês (author)
Other Authors: Coutinho, Maria Francisca (author), Alves, Sandra (author)
Format: conferenceObject
Language:eng
Published: 2018
Subjects:
Mucopolysaccharidosis (MPS)
Mucopolissacaridoses
RNAi
Terapia de Redução do Substrato
Síndrome de Sanfilippo
Doenças Genéticas
Online Access:http://hdl.handle.net/10400.18/5514
Country:Portugal
Oai:oai:repositorio.insa.pt:10400.18/5514

Internet

http://hdl.handle.net/10400.18/5514

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