Molecular Therapy

FIGURE 3

FROM:

Correction of Metabolic, Craniofacial, and Neurologic Abnormalities in MPS I Mice Treated at Birth with Adeno-associated Virus Vector Transducing the Human alpha-L-Iduronidase Gene

Seth D. Hartung, Joel L. Frandsen, Dao Pan, Brenda L. Koniar, Patrick Graupman, Roland Gunther, Walter C. Low, Chester B. Whitley and R. Scott McIvor

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Figure 3.

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Histopathological analysis of tissues for storage material. Group designations are as described for previous figures. One square millimeter, 1-mum-thick sections were stained with toluidine blue, observed at 100times magnification, and scored for pathology as described in Materials and Methods. For liver, heart, and brain, sections from animals 35 and 27 are depicted as examples of uncorrected and corrected pathology, respectively, in the vTRCA1-treated group. In the brain, sections from animals 26 and 45 are depicted as examples of uncorrected and corrected pathology, respectively, in the vTRCA1-treated group. (A–D) Histopathology of liver. H, hepatocyte; Ku, Kupffer cell. Arrows indicate extensive cytoplasmic vacuolization of hepatocytes and Kupffer cells. (E–H) Histopathology of heart. (I–L) Histopathology of lung. (M–P) Histopathology of cerebral cortex. Arrow indicates perivascular cells distended by vacuoles. (Q–T) Histopathology of cerebellum. Arrow indicates large vacuoles in condensed Purkinje cells. (U–X) Histopathology of hippocampus. Arrow indicates perivascular cells distended by vacuoles.

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