Figure 1.
NK1 rescues lysosomal defects and HS accumulation in MPS I, IIIA, and IIIB patient fibroblasts
(A) Human dermal fibroblasts (HDFa) from a healthy control or MPS I, MPS IIIA, and MPS IIIB patient fibroblasts were grown on coverslips and treated or not (mock) with NK1 10−6 M for 48 h before being processed for indirect immunofluorescence. The lysosomal marker LAMP1 (green) and heparan sulfate (pink) were revealed by using specific antibodies. Nuclei (blue) were decorated by DAPI staining. Single focal sections are shown. Images are representative of three independent experiments made in triplicates. Scale bar: 50 μm.
(B and C) Histograms show the quantification relative to the percentage of cells with pathological enlarged lysosomes (green bars) and HS mean value of fluorescence intensity (pink bars). Means ± SEM were obtained from three independent experiments. ∗∗∗ p value <0.001. ns = not significant.