| Project/Area Number |
25670030
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| Research Category |
Grant-in-Aid for Challenging Exploratory Research
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| Allocation Type | Multi-year Fund |
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| Research Field |
Biological pharmacy
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| Research Institution | Hiroshima University |
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Principal Investigator |
SHIMAMOTO Akira 広島大学, 大学院医歯薬保健学研究院, 准教授 (70432713)
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| Co-Investigator(Kenkyū-buntansha) |
KAWAI Hidehiko 広島大学, 原爆放射線医科学研究所, 助教 (30379846)
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| Co-Investigator(Renkei-kenkyūsha) |
YAMAMOTO Takashi 広島大学, 理学研究科, 教授 (90244102)
FURUE Miho 独立行政法人医薬基盤研究所, 細胞資源研究室, 研究リーダー (80257310)
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| Project Period (FY) |
2013-04-01 – 2015-03-31
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| Project Status |
Completed (Fiscal Year 2014)
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| Budget Amount *help |
¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2014: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2013: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
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| Keywords | ウェルナー症候群 / iPS細胞 / 人工ヌクレアーゼ / ゲノム編集 / 遺伝子修正 |
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| Outline of Final Research Achievements |
Werner syndrome (WS) is a rare human autosomal recessive premature aging disorder characterized by early onset of aging-associated diseases, chromosomal instability, and cancer predisposition. Severe symptoms of the disease, such as leg ulcers, cause a significant decline in the quality of life in patients with WS. This study was aimed to establish rejection-free iPS cells whose mutation in WRN gene is corrected by genome editing, and to develop a new therapeutic strategy for WS.
Patient-specific iPS cells were generated and transfected with TALE nuclease vector targeted to the mutated WRN gene and a donor vector designed to replace with the mutated allele, and clones whose WRN mutation was successfully corrected were identified.
These clones will be evaluated that show characteristics equivalent to iPS cells from healthy individuals when differentiated into affected cells in the patient.
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