Experiment: Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
PI: Jason D Heaney, PhD| Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. Detection of editing in non-target tissues. |
| Editing Assay: |
- Fluorescent imaging for tdTomato+ cells - Editing efficiency calculated by counting fluorescent cells (RFP) compared to DAPI stained nuclei |
Organ System Overview