Journal: JCI Insight
Article Title: Modeling skeletal dysplasia in Hurler syndrome using patient-derived bone marrow osteoprogenitor cells
doi: 10.1172/jci.insight.173449
Figure Lengend Snippet: ( A ) IDUA activity in 5-week MPS IH pellets untreated or treated with laronidase (mean ± SEM from 5 MPS IH and 5 MPS IH + rhIDUA pellets): MPS IH, 10.95 ± 4.08 nmol/mg/h; MPS IH + rhIDUA, 290.1 ± 38.72 nmol/mg/h. ** P < 0.01 by paired, 2-sided t test. The red dotted line represents the HD mean. ( B ) Relative GAG content in 5-week MPS IH pellets treated with laronidase compared with untreated (mean ± SEM from 5 MPS IH + rhIDUA compared with 5 untreated pellets): MPS IH + rhIDUA, 0.36 ± 0.11. ** P < 0.01 by paired, 2-sided t test. ( C ) Histological images of Alcian blue– and Sirius red–stained (upper panels) and von Kossa–stained (bottom panels) sections of MPS IH pellets from the same patient untreated or treated with laronidase. Scale bars: 100 μm.
Article Snippet: Pellets treated with the recombinant human L-IDUA were supplemented with 5 mg/mL laronidase (Aldurazyme, Sanofi) for the entire culture period.
Techniques: Activity Assay, Staining