Genome Editing in the Central Nervous System With Engineered Cas9-RNPs
Time: 3:45 pm
day: Day 1 PM
Details:
- Cas9 ribonucleoproteins (RNPs) fused to cationic peptides enables local genome editing in the mouse brain, comparable to delivery with adeno-associated virus 9 (AAV9)
- Treatment with Cas9-RNPs resulted in lower levels of vehicle-specific antibodies and T-cell activation compared to Cas9-AAV9, however, Cas9-RNPs lead to microglial activation that was mitigated by reducing endotoxins/LPS during protein production
- Additional routes of administration and novel formulations of Cas9-RNPs enable further genome editing in the central nervous system
