| Budget Amount *help |
¥17,940,000 (Direct Cost: ¥13,800,000、Indirect Cost: ¥4,140,000)
Fiscal Year 2018: ¥4,810,000 (Direct Cost: ¥3,700,000、Indirect Cost: ¥1,110,000)
Fiscal Year 2017: ¥5,720,000 (Direct Cost: ¥4,400,000、Indirect Cost: ¥1,320,000)
Fiscal Year 2016: ¥7,410,000 (Direct Cost: ¥5,700,000、Indirect Cost: ¥1,710,000)
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| Outline of Final Research Achievements |
By systemic administration of new type of hetero duplex oligonucleotide (HDO), gene knockdown in the brain and spinal cord was observed. Moreover, the effect was enhanced by multiple administration, and we also confirmed that there was dose dependency. Subcutaneous administration was also effective in the central nervous system (CNS). On the other hand, no effect was observed at all by systemic administration of single stranded ASO. So far, gene knockdown effects in CNS by systemic administration of any oligonucleotide have not been demonstrated. We also found that the dynamics of HDO in the blood is significantly different from that of single-stranded ASO. Furthermore, differences in binding proteins and protein binding profiles of HDO in blood were also identified.
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