换一批Intravenous delivery of a chemically modified sulfamidase efficiently reduces heparan sulfate storage and brain pathology in mucopolysaccharidosis IIIA mice.
第一作者：
Susanne,Gustavsson
第一单位：
Research & Translational Science Unit, Swedish Orphan Biovitrum AB (publ), Stockholm, Sweden.
作者：
关键词
ADA, Anti-drug antibodyAF, AutofluorescenceBBB, Blood-brain barrierCHO, Chinese hamster ovarianCM-rhSulfamidase, Chemically modified recombinant human sulfamidaseCNS, Central nervous systemCPM, Chlorpheniramine maleateECL, ElectrochemiluminescenceERT, Recombinant enzyme replacement therapyEnzyme replacement therapyGFAP, Glial fibrillary acidic proteinHS, Heparan sulfateHeparan sulfateLC-MS, Liquid chromatography-mass spectrometryLC-MS/MS, Liquid chromatography-tandem mass spectrometryLIMPII, Lysosomal integral membrane protein IILSD, Lysosomal storage diseaseM6P, Mannose 6-phosphateMPS IIIA, Mucopolysaccharidosis type IIIAMSD-ECL, Meso scale discovery electrochemiluminescenceMTX, MethotrexateMucopolysaccharidosis IIIANeuroinflammationPBS, Phosphate buffered salinePFA, ParaformaldehydePK, PharmacokineticRT, Room temperatureSEC, Size exclusion chromatographySEM, Standard error of meanSanfilippoSulfamidaseTFA, Trifluoroacetic acidWT, Wild type
DOI
10.1016/j.ymgmr.2019.100510
PMID
31528541
发布时间
2024-07-22
- 浏览0
Molecular genetics and metabolism reports
2019年21卷
100510页
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