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Fig. 5. Gaa^-/- diaphragms transduced with rAAV1-CMV-hGAA are free of glycogen deposits. Epon-embedded diaphragms from normal C57B6/129-SvJ (A), untreated Gaa^-/- (B) and Gaa^-/- mice injected with 8.14x10¹⁰ particles of rAAV1-CMV-hGAA in the liver in utero (C) were sectioned and stained with PAS to determine intracellular glycogen content. Electron micrographs were taken of similar tissues from normal (D), untreated (E) and in utero-treated (F) diaphragms to identify glycogen deposits at the cellular level. Representative micrographs from three different vector-treated diaphragms are pictured in C and F. Heart tissue from the rAAV1-CMV-hGAA-treated animal whose diaphragm is pictured in C was stained with PAS to show that other deficient tissues did not benefit from transduction of the diaphragm (I). Heart tissue from normal C57B6/129-SvJ (G) and untreated Gaa^-/- (H) mice was included as controls. Scale bars: 20 µm (A-C,G-I); 500 µm (D-F).

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