2017 Fiscal Year Final Research Report
Amino acid substitution without genetic modification
| Project/Area Number |
15H05491
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| Research Category |
Grant-in-Aid for Young Scientists (A)
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| Allocation Type | Single-year Grants |
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| Research Field |
Bio-related chemistry
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| Research Institution | Nagoya University |
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Principal Investigator |
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| Research Collaborator |
YAMASHITA Shun
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| Project Period (FY) |
2015-04-01 – 2018-03-31
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| Keywords | 革新的遺伝子治療 |
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| Outline of Final Research Achievements |
A nonsense mutation is a genetic mutation that converts a codon coding an amino acid to the stop codon called premature termination codon (PTC). This mutation causes a variety of diseases including Duchenne muscular dystrophy and cystic fibrosis. One of the potential methods for treating such diseases is readthrough therapy that is accomplished by the incorporation of an amino acid to PTC during translation and hence produces the full-length proteins. So far, aminoglycosides and other synthetic compounds that reduce the translation fidelity have been utilized to readthrough therapy. However, their toxicity due to the non-specific induction of inaccurate translation hampered their clinical use. In this study, we have developed a novel readthrough strategy that enables the target PTC-specific readthrough.
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| Free Research Field |
ケミカルバイオロジー
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