Expression of dystrophin via exon skipping with a small chemical

Research Project

Project/Area Number	24390267
Research Category	Grant-in-Aid for Scientific Research (B)
Allocation Type	Partial Multi-year Fund
Section	一般
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)	2012-04-01 – 2015-03-31
Project Status	Completed (Fiscal Year 2014)
Budget Amount *help	¥18,200,000 (Direct Cost: ¥14,000,000、Indirect Cost: ¥4,200,000) Fiscal Year 2014: ¥4,290,000 (Direct Cost: ¥3,300,000、Indirect Cost: ¥990,000) Fiscal Year 2013: ¥4,290,000 (Direct Cost: ¥3,300,000、Indirect Cost: ¥990,000) Fiscal Year 2012: ¥9,620,000 (Direct Cost: ¥7,400,000、Indirect Cost: ¥2,220,000)
Keywords	Duchenne型筋ジストロフィー / エクソンスキッピング / 低分子 / 遺伝子 / ゲノム / 核酸 / 脳神経疾患
Outline of Final Research Achievements	Antisense-meditated exon skipping is the most promising treatment for Duchenne muscular dystrophy. Here, small chemicals that induce dystrophin exon skipping were searched using in vitro splicing system. Unfortunately, chemicals that can be applied for clinical use were not identified yet. Furthermore, a new splicing analysis system that enabled detection of skipping of 10 exons simultaneously was established to enhance screening of chemicals.

Report

(4 results)