Table 1. Comparison of 2-week and 9-week co-administration of gentamicin with NMDI-1 on MPS I-H biochemical endpoints in Idua W392X mouse brain.
% WT Iduronidase Activity | % GAG Reduction | % β-HexReduction | % β-Gluc Reduction | |||||
Treatment | 2wks | 9wks | 2wks | 9wks | 2wks | 9wks | 2wks | 9wks |
Untreated | 0.018% | 0.020% | NA | NA | NA | NA | NA | NA |
Gentamicin | 0.15% | 0.17% | 32% | 49% | 11% | 13% | 35% | 43% |
Gent+NMDI1 | 0.60% | 0.61% | 91% | 75% | 18% | 27% | 56% | 64% |