2016 Fiscal Year Final Research Report
Proof-of-concept of nucleic acid medicine for cardiac channelopahy in an animal model
| Project/Area Number |
15K15049
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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 |
General pharmacology
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| Research Institution | Tokyo Medical and Dental University |
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Principal Investigator |
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| Research Collaborator |
IHARA Kensuke
SASANO Tetsuro
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| Project Period (FY) |
2015-04-01 – 2017-03-31
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| Keywords | 不整脈 |
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| Outline of Final Research Achievements |
Cardiac channelopathy is a main cause of familiar sudden death; because of lack of efficient therapy, the establishment of novel therapeutic strategy is warrated. I have established an animal model of cardiac channelophy by introducing gain-of-function mutant, with use of which I tried to establish a novel therapeutic strategy of cardiac channelopathy. Originally, I planned to reduce gain-of-function mutant gene using oligonucleic acid. However, oligonucleic acid was mainly introduced into the liver, causing liver toxicity as a side action. Then, I changed our plan to use Crispr/Cas9 genome editing strategy. I was sucdeeded in introducing Crispr/Cas9 vector into cardiomyocytes, genome editing in cardiac myocytes, and modulating cardiac ion channel function.
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| Free Research Field |
循環器内科
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