Detalhe da pesquisa
1.
Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB.
J Pharmacol Exp Ther
; 382(3): 277-286, 2022 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-35717448
2.
Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome.
Mol Genet Metab
; 133(2): 185-192, 2021 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33839004
3.
Characterization of disease-specific chondroitin sulfate nonreducing end accumulation in mucopolysaccharidosis IVA.
Glycobiology
; 30(7): 433-445, 2020 07 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-31897472
4.
Cellular Uptake and Delivery of Myeloperoxidase to Lysosomes Promote Lipofuscin Degradation and Lysosomal Stress in Retinal Cells.
J Biol Chem
; 292(10): 4255-4265, 2017 03 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-28115520
5.
Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B.
Mol Ther Methods Clin Dev
; 14: 56-63, 2019 Sep 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-31309128
6.
BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesis.
PLoS One
; 14(1): e0207836, 2019.
Artigo
em Inglês
| MEDLINE | ID: mdl-30657762
7.
Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice.
Mol Ther Methods Clin Dev
; 6: 43-53, 2017 Sep 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-28664165