2015 Fiscal Year Final Research Report
Development of a therapeutic genome editing for hyper-IgE syndrome
| Project/Area Number |
15K15392
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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 |
Pediatrics
|
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| Research Institution | The University of Tokushima |
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Principal Investigator |
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| Project Period (FY) |
2015-04-01 – 2016-03-31
|
| Keywords | 免疫学 / ゲノム編集 |
|---|
| Outline of Final Research Achievements |
Hyper-IgE syndrome is characterized by the extremely high serum IgE levels, severe atopic dermatitis, and recurrent staphylococcal infections to the skin and lung. This disorder is caused by a substitution of a single nucleotide of a single allele of STAT3 gene. To treat this disorder with genome editing, we need to establish a CRISPER/Cas9, which could discriminate a difference of the single nucleotide. To this end, we established a reporter assay, specifically detect double strand break by CRISPER/Cas9. With this model system, we successfully identified a CRISPR/Cas9, which specifically cleave mutant STAT3 allele, but not cleave wild-type STAT3 allele.
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| Free Research Field |
小児科学
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