Antisense oligonucleotide therapy for Fukuyama type congenital muscular dystrophy
| Project/Area Number |
24591510
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | Kobe University |
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Principal Investigator |
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| Co-Investigator(Renkei-kenkyūsha) |
KOBAYASHI Kazuhiro 神戸大学, 大学院医学研究科 分子脳科学, 准教授 (90324780)
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| Project Period (FY) |
2012-04-01 – 2015-03-31
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| Project Status |
Completed (Fiscal Year 2014)
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| Budget Amount *help |
¥5,330,000 (Direct Cost: ¥4,100,000、Indirect Cost: ¥1,230,000)
Fiscal Year 2014: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2013: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2012: ¥2,730,000 (Direct Cost: ¥2,100,000、Indirect Cost: ¥630,000)
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| Keywords | 先天性筋ジストロフィー / 分子標的治療 / RNA創薬 / アンチセンス治療 / 核酸治療 / スプライシング異常 / ドラッグデリバリーシステム / 筋ジストロフィー |
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| Outline of Final Research Achievements |
Fukuyama type congenital muscular dystrophy (FCMD) is a second common, severe childhood muscular dystrophy in Japan. All patients have ancestral insertion of a SINE-VNTR-Alu retrotransposal element (SVA) into a causative gene fukutin. We previously showed that aberrant mRNA splicing, induced by SVA exon-trapping caused FCMD.In this study, we optimized of the best, single AON for clinical trial. We re-designed AONs precisely around the splice sites and assessed the efficacy for exon trap inhibition of these AONs in FCMD patient cells and model mice. By testing on normal Fukutin production and functional analysis, we finally selected one best candidate AON termed AON-F. We also succeeded in improvement on production efficacy for AON-F. We show the promise of splicing modulation therapy as the first radical clinical treatment for FCMD in the near future.
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Report
(4 results)
Research Products
(18 results)
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[Presentation] M. Taniguchi, K. Kobayashi, M. Kanagawa, CC. Yu, T. Oda, A. Kuga, H. Kurahashi, H. O. Akmen, S. DiMauro, T. Yokota, S. Takeda, T. Toda2012
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