A Drosophila High-throughput Drug Screening Platform Identifies Inhibitors Of Misregulated Alternative Splicing Events In Myotonic Dystrophy | 17134
Journal of Biotechnology & Biomaterials
Like us on:
Our Group organises 3000+ Global Conferenceseries Events every year across USA, Europe & Asia with support from 1000 more scientific Societies and Publishes 700+ Open Access Journals which contains over 50000 eminent personalities, reputed scientists as editorial board members.
An automatedin vivo screening platform for the identification of molecules able to modulate alternative splicing events
linked to human disease is introduced. The screening assay is based on the generation of transgenic flies that express a
spliceosensor construct, this is, a minigene whose alternative splicing is coupled to the production of the luciferase reporter.
-based assay we took advantage of the fact that flies were able to closely mirror missplicing events associated
to myotonic dystrophy type 1 (DM1), which is the first described human spliceopathy. The design of the high-throughput
screening pilot study also implemented recent advances in fly assay miniaturization and automation, allowing a top screening
speed of 1,000 compounds per week. Together, more that 15,000 small molecules, which constitutes one of the largest
pharmacological screening to date were screened and identified more than 30 primary hits, several showing promising
anti-DM1 properties according to their putative mechanisms of action and effect on molecular hallmarks of the disease. The
-based tools here described are valuable and flexible resources for innovative drug discovery on human pathologies
originating from alternative splicing misregulation.
Peer Reviewed Journals
Make the best use of Scientific Research and information from our 700 + peer reviewed, Open Access Journals