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Fig. 2 | Orphanet Journal of Rare Diseases

Fig. 2

From: Neural stem cells for disease modeling and evaluation of therapeutics for infantile (CLN1/PPT1) and late infantile (CLN2/TPP1) neuronal ceroid lipofuscinoses

Fig. 2

Effect of recombinant human PPT1/TPP1 protein on reducing enlarged lysosomes in NCL patient NSCs. The western blot analysis (a, b) showed that there is a PPT1 deficiency in PPT1E8/E1 fibroblasts and NSCs, and also there is no TPP1 expression detected in TPP1E4/E6 and TPP1E4/IVS5 fibroblast and NSCs. The treatment of NCL NSCs with 200 nM rPPT1/rTPP1 significantly reduced the LysoTracker dye staining(c), with an effect nearly 99.9% in the NCL NSC lines treated with ERT (d). The images were taken with 40X objective lens. Data are displayed as mean ± SD. ** P < 0.01

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172