2011 Fiscal Year Final Research Report
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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| Keywords | 遺伝子治療学 / 染色体工学 |
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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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