2017 Fiscal Year Final Research Report
Development of molecular therapy for Alport syndrome
| Project/Area Number |
26293203
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Partial Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Kidney internal medicine
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| Research Institution | Kobe University |
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Principal Investigator |
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| Co-Investigator(Kenkyū-buntansha) |
野津 寛大 神戸大学, 医学部附属病院, 助教 (70362796)
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| Co-Investigator(Renkei-kenkyūsha) |
Syono Akemi 神戸大学, 大学院医学研究科, 医学研究員 (10535066)
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| Project Period (FY) |
2014-04-01 – 2018-03-31
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| Keywords | アルポート症候群 / 分子治療 / アンチセンスオリゴヌクレオチド / エクソンスキッピング |
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| Outline of Final Research Achievements |
The purpose of this study is to prove the basic concept of the exon-skipping therapy, a molecular therapy, for male patients with X-linked Alport syndrome, the most popular hereditary nephritis with poor prognosis. The exon-skipping by antisense oligonucleotides will convert severe mutations to milder ones. In this study, we found that antisense oligonucleotides actually induced the exon-skipping in cultured cells obtained from patients and in model mice having the same mutation in the patient. We will apply this therapy to patients with Alport syndrome in the future.
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| Free Research Field |
腎臓学・小児科学
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