| Budget Amount *help |
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2020: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2019: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2018: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
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| Outline of Final Research Achievements |
In recent years, development of nucleic acid drugs such as antisense and siRNA has been progressing, attracting attention as next-generation drugs for hereditary and intractable diseases for which there is no cure. However, the pathways and molecular mechanisms by which nucleic acid drugs are taken up from outside the cell and reach their sites of action inside the cell have not been clarified. In this study, we constructed a screening system to comprehensively search for candidate molecules involved in these pathways using Gapmer-type antisense and splice-switching oligonucleotides, and succeeded in identifying a group of candidate genes.
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