2012 Fiscal Year Final Research Report

Regulation of RNA Splicing by BNA Antisense Oligonucleotides

Research Project

Project/Area Number	23651236
Research Category	Grant-in-Aid for Challenging Exploratory Research
Allocation Type	Multi-year Fund
Research Field	Chemical biology
Research Institution	Osaka University
Principal Investigator	OBIKA Satoshi 大阪大学, 大学院・薬学研究科, 教授 (80243252)
Co-Investigator(Kenkyū-buntansha)	TACHIBANA Keisuke 大阪大学, 大学院・薬学研究科, 助教 (30432446)
Project Period (FY)	2011 – 2012
Keywords	高機能性人工核酸 / BNA / アンチセンス分子 / スプライシング制御 / エクソンスキップ / 筋ジストロフィー / 家族性高コレステロール血症
Research Abstract	First, we established cell lines containing the reporter gene forantisense oligonucleotides that modify the RNA splicing. Next, we designed and analyzed various BNA antisense oligonucleotides using these screening cell lines, which allowed us to obtain several antisense oligonucleotides that induce exon skipping. We also identified three regions in the dystrophin gene as a good target for antisense oligonucleotide-mediated exon skipping.