Presenter: Annemieke Aartsma-Rus, PhD, Leiden University Medical Center
Date: 20 February 2018
Antisense-mediated splicing modulation utilizes antisense oligonucleotides (ASOs) to manipulate the splicing by sterically blocking the binding of splicing factors to pre-mRNA transcripts. While in industry often oligo walks are used to identify the most optimal ASO, generally academic groups do not have the funds to do this and can design only a limited number of ASOs for testing. While ASO design for splicing modulation is partially a trial and error approach, some lessons can be learnt from previous efforts.
Annemieke Aartsma-Rus has designed over 300 ASOs for exon skipping and retrospective analysis has provided her some insights in dos and don’ts with regards to ASO design. In this webinar Annemieke will share this expertise and will outline what to consider when selecting candidate ASOs for splice modulation (e.g. target site selection, thermodynamic properties, checking for off-target effects etc). This presentation will be followed by a Q&A session where you can ask Annemieke your questions during the broadcast.