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Fig. 6 | Orphanet Journal of Rare Diseases

Fig. 6

From: GBA1 as a risk gene for osteoporosis in the specific populations and its role in the development of Gaucher disease

Fig. 6

Reversal of GD symptoms by AAV9-GBA1 gene transfer. A GCase and B CHIT activity was determined in PBMCs of patients with GD (n = 6) compared with age-, sex-, and BMI-matched HDs (n = 6). C Representative histogram of the levels of ER chaperone protein calnexin measured by flow cytometry. PBMCs of patients with GD were treated with AAV9-Ctrl or AAV9-GBA1 (n = 3, independent experiments). Scale bar: 10 μm. D Representative images and E quantification of TRAP-positive polynucleated (≥ 3 nuclei) osteoclasts derived from PBMCs with GD treatment with AAV9-Ctrl or AAV9-GBA1 (n = 3 independent experiments). Transduction of AAV9-Ctrl or AAV9-GBA1 was for 3 days. IC isotype control

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172