Figure 2.

Biochemical evidence of therapeutic effects in immunodeficient Idua^−/− mice after hiNSC transplantation

(A) IDUA enzyme activity quantification (U/mg protein) in brain sections (refer to Figure S1D for further details), brain 1 + 2, brain 3, brain 4, and brain 5 + 6 for unaffected carriers (+/−; n = 6, white), untreated Idua^−/− (−/−; n = 6, black), and transplanted Idua^−/− (−/− Tx; n = 8, gray) cohorts at 8 months post-hiNSC transplantation. Transplanted mice showed higher variability in brain 1 + 2 and brain 3 IDUA activity. Each dot represents the measurement from each animal. (B) β-Hexosaminidase (β-hex) activity quantification (U/mg protein) from the same brain sections in (A). In each brain region examined, β-hex activity is significantly lower in transplanted mice, compared to untreated mice. This decrease suggests functional expression of IDUA by engrafted cells. Data are presented as mean ± SD. Statistical significance is denoted by asterisks (∗p < 0.05; ∗∗p < 0.01; ∗∗∗p < 0.001; ∗∗∗∗p < 0.0001).