When less is actually more: in vitro assessment of the potential of anti-XYLT1 siRNAs to promote substrate reduction in Mucopolysaccharidosis type III

Mucopolysaccharidosis type III (MPS III) refers to a group of four autosomal recessive neurodegenerative lysosomal storage disorders (LSD) caused by the incomplete lysosomal degradation of the heparan sulphate (HS) that accumulates in patient cells and triggers disease. Degeneration of the central n...

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Bibliographic Details
Main Author: Santos, Juliana Inês (author)
Other Authors: Coutinho, Maria Francisca (author), Gaspar, Paulo (author), Prata, Maria João (author), Alves, Sandra (author)
Format: conferenceObject
Language:eng
Published: 2022
Subjects:
Mucopolissacaridose III
Doenças Lisossomais de sobrecarga
Terapias de RNA
Genética Humana
Doenças Genéticas
Online Access:http://hdl.handle.net/10400.18/8144
Country:Portugal
Oai:oai:repositorio.insa.pt:10400.18/8144

Internet

http://hdl.handle.net/10400.18/8144

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