Unlocking Targeted Non-Viral Delivery Methods for RNA-Based Gene Editing Payloads to Minimize Toxic Effects for Improved Therapies
Time: 1:05 pm
day: Pre-Conference Day
Details:
Gene editing payloads contain both the editor and guide RNA, making them much more complex to package. Similarly, there is potential for toxic side effects of permanent off target editing, and such it is vital that drug developers employ cell specific delivery vehicles. Uncover the key principles for successful delivery of RNAbased gene editors to ensure safe and specific editing.
This workshop will address:
- How to sufficiently package the large and complex gene editing machinery?
- How to optimize delivery vehicles for improved cell specificity to minimise the toxic effect of off target gene editing?
- How to successfully scale up non-viral gene editing delivery vehicles with the correct amount of each payload?
- How is endosomal escape affected by the different proportions of cargo component and larger RNAs?