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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Detalhes bibliográficos
Autor principal: Santos, Juliana Inês (author)
Outros Autores: Coutinho, Maria Francisca (author), Gaspar, Paulo (author), Prata, Maria João (author), Alves, Sandra (author)
Formato: conferenceObject
Idioma:eng
Publicado em: 2022
Assuntos:
Mucopolissacaridose III
Doenças Lisossomais de sobrecarga
Terapias de RNA
Genética Humana
Doenças Genéticas
Texto completo:http://hdl.handle.net/10400.18/8144
País:Portugal
Oai:oai:repositorio.insa.pt:10400.18/8144

Texto Completo

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

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