Fluorescent detection of dystrophin mRNA with bispyrene labeled probe

• Project/Area Number: 15K15395
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Pediatrics
• Research Institution: Kobe Gakuin University
• Principal Investigator: Matsuo Masafumi 神戸学院大学, 総合リハビリテーション学部, 教授 (10157266)
• Project Period (FY): 2015-04-01 – 2017-03-31
• Project Status: Completed (Fiscal Year 2016)
• Budget Amount: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000); Fiscal Year 2016: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000); Fiscal Year 2015: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
• Keywords: エクソンスキッピング / ジストロフィン遺伝子 / スプライシング

Outline of Final Research Achievements

Induction of exon skipping of the dystrophin gene is the most promising treatment of Duchenne muscular dystrophy. For this 11 out of 79 dystrophin exons are target of exon skipping. It is the best to induce exon skipping by a small chemical. To search for chemical, in vitro splicing system has been established. However, it takes time and cost to analyze splicing product. In this study it was aimed to establish the method to detect exon skipped mRNA. The validity of this splicing system was confirmed, but it is still on the way to establish efficient detection of skipped mRNA.