Gene therapy of Duchenne muscular dystrophy using own stem cells and human artificial chromosome
| Project/Area Number |
21249022
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| Research Category |
Grant-in-Aid for Scientific Research (A)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Human genetics
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| Research Institution | Tottori University |
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Principal Investigator |
OSHIMURA Mitsuo 鳥取大学, 大学院・医学系・研究科, 教授 (20111619)
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| Co-Investigator(Kenkyū-buntansha) |
OSAKI Mitsuhiko 鳥取大学, 医学部, 准教授 (40325006)
KAZUKI Yasuhiro 鳥取大学, 大学院・医学系・研究科, 助教 (90403401)
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| Project Period (FY) |
2009 – 2011
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| Project Status |
Completed (Fiscal Year 2011)
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| Budget Amount *help |
¥46,410,000 (Direct Cost: ¥35,700,000、Indirect Cost: ¥10,710,000)
Fiscal Year 2011: ¥14,950,000 (Direct Cost: ¥11,500,000、Indirect Cost: ¥3,450,000)
Fiscal Year 2010: ¥14,950,000 (Direct Cost: ¥11,500,000、Indirect Cost: ¥3,450,000)
Fiscal Year 2009: ¥16,510,000 (Direct Cost: ¥12,700,000、Indirect Cost: ¥3,810,000)
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| Keywords | 遺伝子治療学 / 染色体工学 / ヒト人工染色体 / 遺伝子治療 / 筋ジストロフィー / 間葉系幹細胞 / mesoangioblast細胞 |
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| Research Abstract |
Toward gene therapy of Duchenne muscular dystrophy(DMD), we transferred the human artificial chromosome with 2. 4 Mb genomic dystrophin gene(DYS-HAC) to the DMD mouse model(mdx)-derived mesoangioblasts, then the corrected mesoangioblasts were transplanted to mdx-mice. As a result, we succeeded in the long term-amelioration of the dystrophic phenotype in the mdx mice. Thus, a combination of HAC-mediated gene replacement and transplantation with mesoangioblasts will be useful for gene therapy of DMD.
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Report
(4 results)
Research Products
(36 results)
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[Journal Article] Stem cell-mediated transfer of a human artificial chromosome ameliorates muscular dystrophy2011
Author(s)
Tedesco FS, Hoshiya H, D' Antona G, Gerli MF, Messina G, Antonini S, Tonlorenzi R, Benedetti S, Berghella L, Torrente Y, Kazuki Y, Bottinelli R, Oshimura M, Cossu G.
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Journal Title
Sci Transl Med
Volume: Volume 3, number 96
Pages: 96-78
DOI
Related Report
Peer Reviewed
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[Journal Article] Refined human artificial chromosome vectors for gene therapy and animal transgenesis2011
Author(s)
Kazuki Y, Hoshiya H, Takiguchi M, Abe S, Iida Y, Osaki M, Katoh M, Hiratsuka M, Shirayoshi Y, Hiramatsu K, Ueno E, Kajitani N, Yoshino T, Kazuki K, Ishihara C, Takehara S, Tsuji S, Ejima F, Toyoda A, Sakaki Y, Larionov V, Kouprina N and Oshimura M
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Journal Title
Gene The
Volume: Volume 18, number 4
Pages: 384-93
DOI
Related Report
Peer Reviewed
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[Journal Article] Complete genetic correction of iPS cells from Duchenne muscular dystrophy2010
Author(s)
Kazuki Y., Hiratsuka M., Takiguchi M., Osaki M., Kajitani N., Hoshiya H., Hiramatsu K., Yoshino T., Kazuki K., Ishihara C., Takehara S., Higaki K., Nakagawa M., Takahashi K., Yamanaka S. and Oshimura M.
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Journal Title
Mol. Therapy
Volume: Volume18, number 2
Pages: 386-93
DOI
Related Report
Peer Reviewed
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