Date: September 17, 2026
Time: 11-12pm EDT / 5-6pm CEST
Title: Streamlining Exon-Skipping Antisense Oligonucleotide Therapy Development Via a High-Throughput Approach
Description:
Disease-modifying therapies are available for <5% of the >7000 described rare genetic diseases (RGDs). Antisense oligonucleotides (ASOs) represent a promising precision therapy platform for novel RGD therapies, with a leading approach being ASO-mediated ‘skipping’ of an exon containing a deleterious variant to rescue protein function. However, major challenges in this field are that comprehensive analyses identifying exons amenable to ASO-mediated skipping are non-existent, and patient identification and ASO design remain reactive and slow processes. We therefore sought to proactively identify skippable exons across the genome and design ASO sequences that target these exons, shared as an open-access resource.
