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. 2020 Jul 22;21(15):5188. doi: 10.3390/ijms21155188
BBB Blood-Brain Barrier
IDS Iduronate 2-Sulfatase (Elaprase™)
GAG Glycosaminoglycan
LC-MS/MS Liquid Chromatography-Tandem Mass Spectrometry
HS Heparan Sulfate
DS Dermatan Sulfate
CSF Cerebrospinal Fluid
TfR Transferrin Receptor
MPS II Mucopolysaccharidosis II
ETV Enzyme Transport Vehicle
D0A0 4,5 unsaturated uronic acid residue-N-acetyl glucosamine, HS ∆UA-GluNAc
D0S0 4,5 unsaturated uronic acid residue-glucosamine-N-sulfate, ∆UA-GluNS
D0a4 4,5 unsaturated uronic acid residue-N-acetyl galactosamine, 4-O-sulfate, ∆UA-GalNAc4S
HSCT Hematopoietic Stem Cell Transplant
ERT Enzyme Replacement Therapy
Nf-L Neurofilament Light Chain
TfRmu/hu KI Chimeric human/mouse Transferrin Receptor Knock-In mouse