| Budget Amount *help |
¥14,950,000 (Direct Cost: ¥11,500,000、Indirect Cost: ¥3,450,000)
Fiscal Year 2013: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2012: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2011: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2010: ¥4,030,000 (Direct Cost: ¥3,100,000、Indirect Cost: ¥930,000)
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| Outline of Final Research Achievements |
To realize novel protein supplementation therapy for neuronal diseases, we developed an efficient AAV vector production system and vector-producing tumor-targeting cells. By using effective ion-exchange adsorbers, improved GMP production protocol for highly purified vector particles was developed. In addition to the dual ion-exchange procedures, gel filtration chromatography realized ultracentrifugation-free vector purification. As an application for the AAV-mediated protein supplementation therapy, successful transduction of the various disease models was demonstrated. Furthermore, vector-producing tumor-directed stem cells were developed to amplify therapeutic genes.
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