2001 Fiscal Year Final Research Report Summary
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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| 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 |
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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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[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)
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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)
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