期刊
BIOANALYSIS
卷 8, 期 4, 页码 285-295出版社
FUTURE SCI LTD
DOI: 10.4155/bio.15.243
关键词
fit-for-purpose validation; reductive amination; reversed-phase chromatography; stable-isotope labeling; surrogate disaccharides
资金
- Shire
Aims: Heparan sulfate (HS) accumulates in the central nervous system in mucopolysaccharidosis III type A (MPS IIIA). A validated LC-MS/MS assay was developed to measure HS in human cerebrospinal fluid (CSF). Methods & results: HS was extracted and digested and the resultant disaccharides were derivatized with a novel label, 4-butylaniline, enabling isoform separation and isotope-tagged analog introduction as an internal standard for LC-MS/MS. The assay has a LLOQ for disaccharides of 0.1 mu M, +/- 20% accuracy and <= 20% precision. CSF samples from patients with MPS IIIA showed elevated HS levels (mean 4.9 mu M) compared with negative controls (0.37 mu M). Conclusion: This assay detected elevated HS levels in the CSF of patients with MPS IIIA and provides a method to assess experimental therapies.
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