New paper out!

Latest research paper of ESR9, Nuria Suárez Herrera: In this research, we present a proof-of-concept approach to address the high allelic heterogeneity in ABCA4, which is a great challenge in designing therapies for STGD1. This strategy is based on delivering several antisense oligonucleotides embedded in U7snRNAs, providing the opportunity to target various splicing defects using a single therapeutic vector.
The multiple antisense oligonucleotide delivery approach showed potential to rescue several splicing defects, and offers the possibility to target a larger group of STGD1 patients. However, the journey does not end here – more research is crucial to overcome the encountered challenges.