Alan Herbert

Understanding the level of achieved editing efficiency and the functional effect of editing is vital to measure efficacy and understand the true impact of RNA editing therapies. Reliable, reproducible analytical assays are essential in this process and can indicate off-target editing, leading to the development of safer, more efficacious RNA editing medicines.

This workshop will cover:

• Developing robust, reproducible analytical assays that mimic human biological mechanisms to accurately measure levels of editing efficiency

• Utilising functional assays to understand the effect of editing on protein function and disease to elucidate efficacy

• Analysing the transcriptome and genome to understand off-target effects and inform biomarker identification and future therapeutic development