| Budget Amount *help |
¥15,860,000 (Direct Cost: ¥12,200,000、Indirect Cost: ¥3,660,000)
Fiscal Year 2018: ¥3,120,000 (Direct Cost: ¥2,400,000、Indirect Cost: ¥720,000)
Fiscal Year 2017: ¥3,120,000 (Direct Cost: ¥2,400,000、Indirect Cost: ¥720,000)
Fiscal Year 2016: ¥3,120,000 (Direct Cost: ¥2,400,000、Indirect Cost: ¥720,000)
Fiscal Year 2015: ¥3,120,000 (Direct Cost: ¥2,400,000、Indirect Cost: ¥720,000)
Fiscal Year 2014: ¥3,380,000 (Direct Cost: ¥2,600,000、Indirect Cost: ¥780,000)
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| Outline of Final Research Achievements |
The specific characteristics of the recombinant adeno-associated virus (rAAV) with safety and long-term expression have made it an attractive transduction tool for clinical gene therapy of neuromuscular diseases. However, in vivo gene transduction with the rAAV depends upon laborious procedures for the production of the vector stocks to meet end-product specifications. We developed methods of producing rAAV with scalable purification using the high-performance ion exchange membrane adsorbers for considerable in vivo experimentation and clinical investigation. We adopted our production system to investigate AAV vector-mediated ex vivo MSC transduction strategy for the treatment of various neuromuscular diseases including stroke. Furthermore, vector-producing MSC would be promising to realize tumor-targeting as well as transgene amplification in situ.
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