Study on treatment of Duchenne muscular dystrophy by inducing exon skipping
Project/Area Number |
19390284
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Research Category |
Grant-in-Aid for Scientific Research (B)
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Allocation Type | Single-year Grants |
Section | 一般 |
Research Field |
Pediatrics
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Research Institution | Kobe University |
Principal Investigator |
MATSUO Masafumi Kobe University, 大学院医学研究科, 教授 (10157266)
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Co-Investigator(Kenkyū-buntansha) |
TAKESHIMA Yasuhiro 神戸大学, 大学院医学研究科, 准教授 (40281141)
YAGI Mariko 神戸大学, 大学院医学研究科, 助教 (60362787)
|
Project Period (FY) |
2007 – 2008
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Project Status |
Completed (Fiscal Year 2008)
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Budget Amount *help |
¥18,850,000 (Direct Cost: ¥14,500,000、Indirect Cost: ¥4,350,000)
Fiscal Year 2008: ¥7,020,000 (Direct Cost: ¥5,400,000、Indirect Cost: ¥1,620,000)
Fiscal Year 2007: ¥11,830,000 (Direct Cost: ¥9,100,000、Indirect Cost: ¥2,730,000)
|
Keywords | 小児神経学 / ジストロフィン / リボゾーム / エクソンスキッピング / 治療 |
Research Abstract |
私達のデュシェンヌ型筋ジストロフィー(DMD)の治療結果により、DMD骨格筋では「タンパク合成システムの早期破綻」が想定された。しかし、リボソームRNAのサイズの分布には年齢による明確な違いは見い出せなかった。一方、リボソームで利用されるジストロフィンmRNAの量を決定するnonsense mediated RNA decayの年齢差あるいは、核タンパクの寿命ならびに挙動が「タンパク合成システムの早期破綻」に結びついている可能性も示唆された。
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Report
(3 results)
Research Products
(18 results)
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[Journal Article] In vitro splicing analysis reveals that availability of a cryptic splice site is not a determinant for alternative splicing patterns caused by +1G>A mutations in introns of the dystrophin gene.2008
Author(s)
Habara, Y.,Takeshima, Y., Awano, H., Okizuka, Y., Zhang, Z., Saiki, K., Yagi, M., Matsuo, M.
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Journal Title
J Med Genet. in press.
Pages: 0-0
Related Report
Peer Reviewed
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[Journal Article] Wide ranges of serum myostatin concentrations in Duchenne muscular dystrophy patients.2008
Author(s)
Awano, H., Takeshima, Y., Okizuka, Y., Saiki, K., Yagi, M., Matsuo, M.
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Journal Title
Clin Chim Acta. 391
Pages: 115-117
Related Report
Peer Reviewed
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[Journal Article] Dystrophin nonsense mutations can generate alternative rescue transcrips in lymphocytes.2008
Author(s)
Nisiyama, A., Takeshima, Y., Zhang, Z., Habara, Y., Tran, TH., Yagi, M., Matsuo, M.
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Journal Title
Ann Hum Genet. 72
Pages: 717-724
Related Report
Peer Reviewed
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[Journal Article] High incidence of electrocardiogram abnormalities in young patients with duchenne muscular dystrophy.2008
Author(s)
Takami, Y., Takeshima, Y., Awano, H., Okizuka, Y., Yagi, M. and Masuo, M.
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Journal Title
Pediatr. Neurol. 39
Pages: 399-403
Related Report
Peer Reviewed
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[Journal Article] A 2-bp deletion in exon 74 of the dystrophin gene does not clearly induce muscle weakness.2008
Author(s)
Kimura, S. Ito, K. Ueno, H. Ikezawa, M. Takeshima,Y. Yoshioka, K.Ozasa, S. Nakamuara, K. Nomura, K. Matsukura, M. Mitsui, K. Matsuo, M. Miike, T.
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Journal Title
Brain & Development. 18
Pages: 672-677
NAID
Related Report
Peer Reviewed
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[Journal Article] Multi-exon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne Muscular Dystrophy.2007
Author(s)
Beroud C, Giraud TS, Matsuo M, Hamroun D., Humbertclaude V, Monnier N, Moizard MP, Voelckel MA, Calemard LM, Boisseau P, Blayau M, Philippe C, Cossee M, Pages M, Rivier F, Danos O, Garcia L, Claustres M.
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Journal Title
Hum Mutat. 28
Pages: 196-202
Related Report
Peer Reviewed
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