Establishment of treatment of Duchenne muscular dystrophy
| Project/Area Number |
12557068
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Single-year Grants |
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| Section | 展開研究 |
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| Research Field |
Pediatrics
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| Research Institution | Kobe University |
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Principal Investigator |
MATSUO Masafumi Kobe University, School of Medicine, Professor, 医学部, 教授 (10157266)
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| Co-Investigator(Kenkyū-buntansha) |
TAKESHIMA Yasuhiro Kobe University, Hospital, Assistant, 医学部・附属病院, 助手 (40281141)
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| Project Period (FY) |
2000 – 2001
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| Project Status |
Completed (Fiscal Year 2001)
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| Budget Amount *help |
¥13,400,000 (Direct Cost: ¥13,400,000)
Fiscal Year 2001: ¥3,600,000 (Direct Cost: ¥3,600,000)
Fiscal Year 2000: ¥9,800,000 (Direct Cost: ¥9,800,000)
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| Keywords | Duchenne muscular dystrophy / splicing / splicing enhancer sequence / exon skipping / antisense oligonucleotide / スキッピング / アンチセンス / スプライシング促進配列 / スプライシング |
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| Research Abstract |
It has been proposed that Duchenne muscular dystrophy can be treated with modification of out-of-frame mutation into in-frame mutation by inducing exon skipping. In this study the possibility to treat DMD was evaluated by seeing inducibility of exon skipping. In the previous study we showed exon 19 skipping can be induced by blocking the function of splicing enhancer sequence with antisense oligonucleotides. To apply this strategy we looked for splicing enhancer sequence in exons that are located in deletion hot spots. From exon sequence purine-rich sequence were extracted as candidate sequences for splicing enhancer sequence and splicing enhancer activity of those candidate sequences were analyzed by using Drosophilla dsx minigene. Thereby some antisense oligonucleotides were found to have an ability to induce exon skipping.
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Report
(3 results)
Research Products
(18 results)
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[Publications] Suminaga, R, Takeshima, Y, Yasuda, K, Shiga, N, Nakamura, H, Matsuo, M.: "Non-homologous recombination between Alu and LINE- 1 repeats caused a 430 kbdeletion of the dystrophin gene : A novel source of genomic instability"J. Hum. Genet.. 45. 331-336 (2000)
Description
「研究成果報告書概要(欧文)」より
Related Report
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[Publications] Takeshima, Y, Yagi, M, Ishikawa, Y, Ishikawa, Y, Minami, T, Nakamura, H, Matsuo, M.: "Oligonucleotides against a splicing enhancer sequence led to dystrophin production in muscle cells from a Duchenne muscular dystrophy patient"Brain Dev. 23. 788-798 (2001)
Description
「研究成果報告書概要(欧文)」より
Related Report
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