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).