| Budget Amount *help |
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2010: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2009: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2008: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
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| Research Abstract |
To develop safe and effective therapy inhibiting complement mediated hemolysis in paroxysmal nocturnal hemoglobinuria (PNH), high affinity nuclease-resistant RNA aptamers that bind specifically to C8 or C9 were identified using SELEX methodology. C8-4-101 aptamer clone bound C8 with a Kd of 15nM and a Bmax of 74% and C9-16-8 aptamer clone bound C9 with a Kd of 3nM and a Bmax of 67%, with inhibiting hemolytic activity similar to or greater than control (C5 aptamers). The development of blocking RNA aptamers, using the SELEX procedure against terminal complement proteins C8 or C9, represents a novel potential therapeutic option for PNH.
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