Experiment: FUS (focused ultrasound) array validation in Ai9 mice
PI: Kam W Leong, PhD
Description: 9.3 week-old Ai9 mice (4 male and 4 female) were administered Ai9-targeting SaCas9 AAV9 vector through intravenous adminsitration (2E12 vg/mouse) and left hemisphere was targeted by FUS (focused ultrasound) array for BBB (blood brain barrier) opening
Editing Assay:
- Edited volume: summation of total TdTomato+ area from serial sections or lightsheet detection. 1 pair analyzed in house using IHC (immunohistochemistry) /confocal; 1 pair analyzed by The Jackson Laboratory and 2 pairs analyzed using lightsheet microscope
- Neuron editing efficiency at ROI (region of interest) (1200um^2) calculated by counting TdTomato+ compared with NeuN+ (neuron) cells. 1 pair analyzed in house using IHC (immunohistochemistry)/confocal; 1 pair analyzed by The Jackson Laboratory and 2 pairs analyzed using lightsheet microscope
Parent Project: Focused Ultrasound-mediated Delivery of Gene-editing Elements to the Brain for Neurodegenerative Disorders
Download: Submitted files
Publications:
- Focused ultrasound-mediated brain genome editing. NCBI
Results |
| Editing Efficiency | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Condition | Tissue | Cell Type | Sex | Editor | Model | Delivery | Target Locus | Guide | Vector | Dosage | Injection Frequency | Signal | Image |
| Male subjects | brain (TARGET) | neuron | Male | SaCas9 | Ai9 mouse | AAV+Focused Ultrasound | Ai9/Ai14 reporter transgene | Sa_Ai9_L; Sa_Ai9_R | Ai9LR21-SaCas9 | 2E12 vg/mouse | once |
 |
|
| Male subjects | brain (TARGET) | Male | SaCas9 | Ai9 mouse | AAV+Focused Ultrasound | Ai9/Ai14 reporter transgene | Sa_Ai9_L; Sa_Ai9_R | Ai9LR21-SaCas9 | 2E12 vg/mouse | once | |||
| Female subjects | brain (TARGET) | neuron | Female | SaCas9 | Ai9 mouse | AAV+Focused Ultrasound | Ai9/Ai14 reporter transgene | Sa_Ai9_L; Sa_Ai9_R | Ai9LR21-SaCas9 | 2E12 vg/mouse | once |
 |
|
| Female subjects | brain (TARGET) | Female | SaCas9 | Ai9 mouse | AAV+Focused Ultrasound | Ai9/Ai14 reporter transgene | Sa_Ai9_L; Sa_Ai9_R | Ai9LR21-SaCas9 | 2E12 vg/mouse | once | |||
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