Fig. 2

Small intestinal histopathology in MNGIE mice. a, b Hematoxylin-Eosin (H&E) stains of the small intestine of young (2-month-old) control mice (a) and age-matched Tymp^−/-Upp1^−/− mutants (b) show normally layered organization of the intestinal wall in both groups. c, d H&E stains of the small intestine show normal thickness of the tunica muscularis propria in old (12-month-old) control mice (c), whereas in age-matched Tymp^−/-Upp1^−/− mutants (d) the muscle wall is atrophic. e Bone marrow cell chimerism and vector copy number in recipients of 0.5 × 10⁶ Lin- cells transduced by LV-PGK-TP (MOI10) (n = 3 mice). f Quantification of Thd and d-Urd in urine of untreated controls and age-matched recipients 6 and 11 months after transplantation (n = 3 mice). g H&E stain of the small intestine shows that atrophy of the tunica muscularis propria is prevented in old (12-month-old) Tymp^−/-Upp1^−/− mice 10 months after treatment. h Quantification confirms atrophy of the muscle wall in 12-month-old Tymp^−/-Upp1^−/− mice compared to wild-type age-matched controls. Treatment is associated with normal thickness of the tunica muscularis propria. i Quantification of the number of myenteric ganglion cell groups per tissue section shows progressive loss of ganglion cells in Tymp^−/-Upp1^−/− mice, without effect of the treatment. N = 2–4 mice/group; in all graphs lines represent the median; *P < 0.05, **P < 0.01, ***P < 0.001. Original magnification (a-d and g): 200x