| Budget Amount *help |
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2019: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2018: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2017: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
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| Outline of Final Research Achievements |
We have been conducting research and development of enzyme replacement therapy to continuously supply defective proteins in patients. We focus on pre-adipocytes prepared from adipose tissue as target cells, where we introduce therapeutic genes ex vivo to secrete therapeutic proteins (adipocytes for gene therapy) and transplant into patients. In this study, we aimed to develop a transport solution that can preserve adipocytes for gene therapy in the non-freezing state for at least 48 to 72 hours, so that they can be administered to a base hospital near the patient. As a result, we have established a cell preservation solution to keep viability of gene-transduced pre-adipocytes at 4 ° C for at least 48 hours. Based on the obtained basic data, it is planned to be put to practical use through safety tests such as cell characteristic tests and non-clinical animal tests.
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