Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder?

Mucopolysaccharidosis type II (Hunter Syndrome) is a rare, x-linked recessive, progressive, multi-system, lysosomal storage disease caused by the deficiency of iduronate-2-sulfatase (IDS), which leads to the pathological storage of glycosaminoglycans in nearly all cell types, tissues and organs. The...

Descripción completa

Detalles Bibliográficos
Autores principales: Horgan, Claire, Jones, Simon A., Bigger, Brian W., Wynn, Robert
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9105950/
https://www.ncbi.nlm.nih.gov/pubmed/35563245
http://dx.doi.org/10.3390/ijms23094854

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9105950/
https://www.ncbi.nlm.nih.gov/pubmed/35563245
http://dx.doi.org/10.3390/ijms23094854

Ejemplares similares