Intramuscular injection of AAV2.1-TFEB in GAA −/− mice promotes glycogen clearance and attenuates PD pathology.
A. Glycogen assay in TFEB-injected gastrocnemii and in the contralateral muscles. In TFEB-injected muscles glycogen levels were significantly decreased compared to those in untreated muscles. * indicates statistically significant differences (p = 1.0 × 10−4; n = 6; Student's t-test).
B. PAS staining of TFEB-treated muscle shows a reduction of lysosomal glycogen stores (puncta) compared to those in untreated muscle. Original magnification: 20×.
C. LAMP1 staining of TFEB-injected gastrocnemii and of the contralateral untreated muscles. In TFEB-treated muscles, the size of LAMP1-positive vesicles was reduced. Bar: 2 µm.