Keywords: Lysosomal storage disorder; MPS IIIA; Mouse; Cerebrospinal fluid; Enzyme replacement therapy; Osmotic pump;
مقالات ISI (ترجمه نشده)
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A phase 1/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA
Keywords: AE; adverse event; BSID-III; Bayley Scales of Infant and Toddler Development, Third Edition; CSF; cerebrospinal fluid; CNS; central nervous system; DQ; developmental quotient; EC; Enzyme Commission; ELISA; enzyme-linked immunosorbent assay; GAG; glycosami
Determination of the role of injection site on the efficacy of intra-CSF enzyme replacement therapy in MPS IIIA mice
Keywords: Lysosomal storage disorder; Sanfilippo syndrome; MPS IIIA; Mouse; Cerebrospinal fluid; Enzyme replacement therapy;
Acquired Autistic Behaviors in Children with Mucopolysaccharidosis Type IIIA
Keywords: ADOS; Autism Diagnostic Observation Schedule; ASD; Autism spectrum disorder; MPS IIIA; Mucopolysaccharidosis type IIIA;
Development of cerebellar pathology in the canine model of mucopolysaccharidosis type IIIA (MPS IIIA)
Keywords: GAD65/67; glutamic acid decarboxylase 65/67; GlcNS-UA; glucosamine-N-sulfate-uronic acid; INCL; infantile neuronal ceroid lipofuscinosis; LC-ESI-MS/MS; liquid chromatography-electrospray ionization tandem mass spectrometry; LSD; lysosomal storage diso
Exocytosis is impaired in mucopolysaccharidosis IIIA mouse chromaffin cells
Keywords: CYWHS; Children Youth and Women's Health Service; DMEM; Dulbecco's modified Eagle medium; DMF; dimethylformamide; GlcNS-UA; heparan sulphate-derived disaccharide glucosamine N-sulphate [α-1,4] uronic acid; Hepes; 4-(2-hydroxyethyl)-1-piperazineethane
Neuroinflammatory and oxidative stress phenomena in MPS IIIA mouse model: The positive effect of long-term aspirin treatment
Keywords: MPS IIIA; Mucopolysaccharidosis (MPS) type IIIA, Sanfilippo disease type A; LSD; Lysosomal storage diseases; GAGs; Glycosaminoglycans; CNS; Central nervous system; GFAP; Glial fibrillary acidic protein; NSAIDs; Nonsteroidal anti-inflammatory drugs; PCR; P
Impact of high-dose, chemically modified sulfamidase on pathology in a murine model of MPS IIIA
Keywords: MPS IIIA; Mucopolysaccharidosis IIIA; CNS; central nervous system; LSD; lysosomal storage disorder; SGSH; N-sulfoglucosamine sulfohydrolase; HS; heparan sulfate; ERT; enzyme replacement therapy; M6P; mannose-6-phosphate; M6PR; mannose-6-phosphate receptor
Effect of cisternal sulfamidase delivery in MPS IIIA Huntaway dogs-A proof of principle study
Keywords: MPS IIIA; mucopolysaccharidosis type IIIA; LSD; lysosomal storage disorder; SGSH; sulfamidase; CSF; cerebrospinal fluid; rhSGSH; recombinant human sulfamidase; HS; heparan sulfate; ERT; enzyme replacement therapy; CM; cerebellomedullary cistern; HPLC; hig
Lentiviral-mediated gene therapy for murine mucopolysaccharidosis type IIIA
Keywords: HIV-1; Vector; MPS IIIA; Mice; Correction
Injection of recombinant human sulfamidase into the CSF via the cerebellomedullary cistern in MPS IIIA mice
Keywords: MPS IIIA; Lysosomal storage disorder; Mouse; CNS; Recombinant enzyme; Cerebrospinal fluid; Mass spectrometry; Neuropathology; Behavior; Humoral immune response