2010 Fiscal Year Final Research Report
Development of target therapeutic for PNH using RNA aptamer
| Project/Area Number |
20591123
|
|---|
| Research Category |
Grant-in-Aid for Scientific Research (C)
|
| Allocation Type | Single-year Grants |
|---|
| Section | 一般 |
|---|
| Research Field |
Hematology
|
|---|
| Research Institution | Osaka University |
|---|
Principal Investigator |
NISHIMURA Jun-Ichi Osaka University, 医学系研究科, 助教 (80464246)
|
|---|
| Co-Investigator(Kenkyū-buntansha) |
KANAKURA Yuzuru 大阪大学, 医学系研究科, 教授 (20177489)
|
|---|
| Project Period (FY) |
2008 – 2010
|
| Keywords | 発作性夜間ヘモグロビン尿症(PNH) / RNAアプタマー / 補体 / 溶血 / SELEX |
|---|
| 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.
|
Research Products
(36 results)
