![Fig. 1. Survival of treated and untreated Sandhoff mice. / (A) Mice (n = 4-6 per group) were monitored daily, and a humane endpoint was applied when they became moribund and unable to right themselves when laid on their sides. In all the treated groups, the increase in survival relative to SHUT was statistically significant (P < .001). The synergistic effect was estimated using Bootstrap method. Synergy quotient (SQ) was defined asSQ = [(SHBMT − SHUT) + (SHNB − DNJ − SHUT)]/(SHBMT/NB − DNJ − SHUT), where SHUT,SHBMT,SHNB − DNJ, andSHBMT/NB − DNJare the mean survival of the Sandhoff mice untreated, treated with BMT, treated with NB-DNJ, and treated with BMT andNB-DNJ, respectively. If there is synergy, the SQ is less than one. (B) Analysis of β-hexosaminidase activity in nerve tissues and (C) in visceral organs of the Sandhoff mice after BMT treatment. Each organ was assayed in triplicate. The means for the different animals were averaged (± SEM) and expressed as a percentage of wild-type levels. n = 4 to 6 animals per group.](https://ash.silverchair-cdn.com/ash/content_public/journal/blood/97/1/10.1182_blood.v97.1.327/6/h80110544001.jpeg?Expires=1724941626&Signature=e9LEhKmvXVp6H7qJtzrGM02lLonRU5j96om12OVbbyEco0TbEG-bA120nAKcSUX-fagUxSs3sPlglF5GlsH3T9sh2upKv1ef6~USyh4Afh5lWu-k7gnuXpQC1P0HAs3xGdjeeDVhZEYSE2NyzBLrgN1prJ8iy~Y1qYm0TeML8-7z0wt8wQsA~pkhbpxSZhWd9tqRr6QIogmAbMwHBnekoBRbIraBZX88CL2XbUVi0EVD170N5U99dOw3NOdBwSlLCNEsWM1-AADk5weAnOBOeVLfGmbg~5hK05LQUJPt9rHQyjxrSVjo-88lNXsLlT5ZHaO36Fswb3jYb6E2fd5NTw__&Key-Pair-Id=APKAIE5G5CRDK6RD3PGA)
Survival of treated and untreated Sandhoff mice.
(A) Mice (n = 4-6 per group) were monitored daily, and a humane endpoint was applied when they became moribund and unable to right themselves when laid on their sides. In all the treated groups, the increase in survival relative to SHUT was statistically significant (P < .001). The synergistic effect was estimated using Bootstrap method. Synergy quotient (SQ) was defined asSQ = [(SHBMT − SHUT) + (SHNB − DNJ − SHUT)]/(SHBMT/NB − DNJ − SHUT), where SHUT,SHBMT,SHNB − DNJ, andSHBMT/NB − DNJare the mean survival of the Sandhoff mice untreated, treated with BMT, treated with NB-DNJ, and treated with BMT andNB-DNJ, respectively. If there is synergy, the SQ is less than one. (B) Analysis of β-hexosaminidase activity in nerve tissues and (C) in visceral organs of the Sandhoff mice after BMT treatment. Each organ was assayed in triplicate. The means for the different animals were averaged (± SEM) and expressed as a percentage of wild-type levels. n = 4 to 6 animals per group.
