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. 2023 Jul 1;31(9):2651–2661. doi: 10.1016/j.ymthe.2023.06.018

Figure 2.

Figure 2

Increased lysosomal exocytosis in PiZ livers after TRPML1 gene transfer

(A) Representative images of immunofluorescence for LAMP1 and TRPML1-Myc in livers of PiZ mice harvested 4 weeks after AAV injections. (B) Representative images of immunofluorescence for LAMP1 and polymeric ATZ in livers of PiZ mice harvested 4 weeks after AAV injections. The dashed lines are superimposed to the margins of the plasma membranes to highlight single cells. (C) Representative electron microscopy images of PiZ livers injected with AAV-GFP or AAV-TRPML1 and harvested either 4 or 12 weeks post injection showing lysosomes (white arrows) close to the plasma membrane shown in red. The relative quantifications of lysosomes close to the plasma membrane is shown (n = 3 per group). (D) Triple immunostaining for the lysosome marker LAMP1, the plasma membrane marker laminin 2, and the basolateral membrane marker Na-K ATPase on livers of PiZ mice injected with AAV-GFP or AAV-TRPML1 and harvested at 4 weeks post injection. Quantification of LAMP1/laminin 2 co-localization is shown (n = 3 per group; n = 4 fields/sample). (E) Serum IDS and GALNS activities in AAV-GFP- and AAV-TRPML1-injected PiZ mice at 2 weeks post injection (IDS, n = 6 per group; GALNS, n = 4 for AAV-GFP; and n = 6 per AAV-TRPML1). Data are shown as mean ± standard error. Student’s t test: ∗p value < 0.05; ∗∗p value < 0.01. Abbreviations are as follows: BC, bile canaliculi; lyso, lysosomes; PM, plasma membrane.