Cas & Non-Cas Proteins for RNA Modulation: RNA Editing & Beyond

Time: 11:30 am
day: Day One


• Overview of naturally-occurring Cas proteins, as well as synthetic, non- Cas proteins engineered to target and modulate RNA transcripts
• Expand on RNA binding proteins and effector domains designed to target disease-causing RNA, with a particular focus on the following therapeutic modalities: destroying dysfunctional RNA, destroying dysfunctional RNA and replacing it with a normal copy, enhancing RNA translation to produce more of a specific protein, modifying splicing, and RNA editing
• Explore the therapeutic applications of the versatile RNA targeting and RNA modification mechanisms in addressing neuromuscular, neurodegenerative and ophthalmological diseases