| Budget Amount *help |
¥5,330,000 (Direct Cost: ¥4,100,000、Indirect Cost: ¥1,230,000)
Fiscal Year 2014: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2013: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2012: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
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| Outline of Final Research Achievements |
We examined the feasibility of adeno-associated viral serotype-9 (AAV9)-mediated systemic neonatal gene therapy of metachromatic leukodystrophy (MLD) mice to treat central nervous system (CNS) disorders. AAV9 vector expressing human arylsulfatase A (AAV9/ASA) was injected into the jugular vein of newborn MLD mice. Global gene transfer into the brain, across the blood brain barrier, and long-term ASA expression in the CNS were detected in treated mice. Significant inhibition of the accumulation of sulfatide (Sulf) in the brain was confirmed by Alcian blue staining and biochemical analysis of the Sulf content. In a behavior test, treated mice showed a greater ability to traverse narrow balance beams than untreated mice. These data clearly demonstrate that MLD mice can be effectively treated through neonatal systemic injection of AAV9/ASA. Thus, AAV9 mediated systemic neonatal gene therapy is useful to treat various CNS disorders.
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