FIGURE 2
FROM:
Central Nervous System-directed AAV2/5-Mediated Gene Therapy Synergizes with Bone Marrow Transplantation in the Murine Model of Globoid-cell Leukodystrophy
Darshong Lin, Anthony Donsante, Shannon Macauley, Beth Levy, Carole Vogler and Mark S Sands
BACK TO ARTICLEFigure 2.
GALC activity. GALC activity, expressed as a percent normal (% normal), was measured in the brain, liver, and kidney of Twitcher mice following individual or combined therapies. The normal GALC levels were determined by the mean activity in each tissue measured in three normal animals at 38 days of age. Untreated Twitcher mice (Twi, 38 days of age, n=3) had 2.2, 0, and 0.9% GALC activity in the brain, liver, and kidney, respectively. Twitcher animals receiving BMT alone (BMT, 34–50 days of age, n=5) had GALC levels in the brain that were not significantly different from untreated Twitcher animals. However, BMT-treated animals had 6.5 and 0.6% normal levels in the liver and kidney, respectively. Intracranial injections of AAV2/5 alone (AAV2/5, 34 days of age, n=3) resulted in 217, 4.8, and 1.0% normal GALC levels in the brain, liver, and kidney, respectively. The combination-treated twitcher mice (AAV/BMT, 34 days of age, n=3) had 380, 29, and 2.6% normal GALC levels in the brain, liver, and kidney, respectively. The bars represent the mean
1SEM.
