Neurological disease modeling using pluripotent and multipotent stem cells: a key step towards understanding and treating mucopolysaccharidoses
S Carvalho, JI Santos, L Moreira, M Gonçalves… - Biomedicines, 2023 - mdpi.com
Despite extensive research, the links between the accumulation of glycosaminoglycans
(GAGs) and the clinical features seen in patients suffering from various forms of …
(GAGs) and the clinical features seen in patients suffering from various forms of …
A novel CRISPR/Cas9-based iduronate-2-sulfatase (IDS) knockout human neuronal cell line reveals earliest pathological changes
L Badenetti, R Manzoli, M Trevisan, F D'Avanzo… - Scientific Reports, 2023 - nature.com
Multiple complex intracellular cascades contributing to Hunter syndrome (
mucopolysaccharidosis type II) pathogenesis have been recognized and documented in the …
mucopolysaccharidosis type II) pathogenesis have been recognized and documented in the …
Gene-modified neural progenitor cells for the treatment of neuropathic lysosomal storage diseases
O Mandolfo, BW Bigger - Neural Regeneration Research, 2023 - journals.lww.com
Lysosomal storage diseases: Lysosomal storage diseases (LSDs) are a family of about 70
disorders, with an overall incidence of 1: 7000 live births. They are caused by dysfunctional …
disorders, with an overall incidence of 1: 7000 live births. They are caused by dysfunctional …
[PDF][PDF] MODELING LYSOSOMAL STORAGE DISORDERS IN AN INNOVATIVE WAY: ESTABLISHMENT AND CHARACTERIZATION OF STEM CELL CULTURES …
SP Carvalho - 2023 - estudogeral.uc.pt
Nacional de Saúde Dr. Ricardo Jorge, me integraram como se já ali pertencesse há muito.
Por toda a convivência e diálogo diários (por vezes difícil, eu sei), pela disponibilidade de …
Por toda a convivência e diálogo diários (por vezes difícil, eu sei), pela disponibilidade de …
[HTML][HTML] Study of the neurodegenerative process in Mucopolysaccharidosis type VII and its correction by gene therapy
AO Salgado - 2023 - dialnet.unirioja.es
La mucopolisacaridosis tipo VII es un trastorno autosómico recesivo causado por una
deficiencia de β-glucuronidasa,(GUSB) que conduce a la acumulación de …
deficiencia de β-glucuronidasa,(GUSB) que conduce a la acumulación de …
[HTML][HTML] Study of the neurodegenerative process in Mucopolysaccharidosis type VII and its correction by gene therapy
A Onieva Salgado - 2023 - ddd.uab.cat
La mucopolisacaridosis tipus VII és un trastorn autosòmic recessiu causat per una
deficiència de β-glucuronidasa,(GUSB) que condueix a l'acumulació de glicosaminoglicans …
deficiència de β-glucuronidasa,(GUSB) que condueix a l'acumulació de glicosaminoglicans …