Advancing RNAi experiments towards in vivo studies represents a more rigorous level of testing for nucleic acid therapeutics beyond in vitro, cell-based experiments. Dr Garrett Rettig presents considerations for producing significant knockdown, including:
- A suitable model system for testing gene knockdown or phenotypic effect
- Route of delivery
- Delivery vehicle
- Potential for off-target effects
Then, Pascal Tétreault presents an example of an in vivo RNAi application, along with a description of the logistics of in vivo experiments conducted in Dr Philippe Sarret's pre-clinical pain lab. The lab uses DsiRNA silencing technology to study the mechanisms of action of G protein-coupled receptors involved in chronic pain in the spinal cord.
Both Dr Rettig and Tétreault then discuss challenges for in vivo RNAi experimental setup and take questions from the audience.