Development of Targeted LNP for Efficient In Vivo Editing of Hematopoietic Stem Cells
Time: 3:45 pm
day: Conference Day One
Details:
- Designing LNP composition to de-target from liver and redistributed to target organs of interest
- Developing screening platform to select targeting ligands that maximize the internalization of cell-surface targets while maintaining cellular specificity for HSCs
- Conjugating the improved targeting ligands on the optimal liver de-targeting base LNP achieved efficient editing of HSPCs in humanized mice as well as efficient editing in HSCs in NHPs after a single dose
