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ExperimentFUS (focused ultrasound) array validation in Ai9 mice
Experiment ConditionFemale subjects
Assay Description: 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
Tissue Measured:brain
Cell Type :neuron
Measurement Type:Editing Efficiency
Record ID15000001500

EditorSaCas9
Delivery SystemAAV+Focused Ultrasound
Delivery System SubtypeViral vector
GuideSa_Ai9_L; Sa_Ai9_R
VectorAi9LR21-SaCas9;

Model SpeciesMouse
Model NameB6.Cg-Gt(ROSA)26Sor ^ tm9(CAG-tdTomato)Hze/J

Application MethodIV with focused ultrasound (FUS)
Application Sitesystemic, left brain hemisphere
Dosage2E12 vg/mouse
Injection Frequencyonce
Injection Rate
Injection Volume100 microliters
Days post injection3 weeks
Editor FormatAAV vector encoded
Antidote Id
Antidote Description

Measured Values
Samples Size: 4
 
Replicate Result (Editing Efficiency)
Measurement Units: Signal
Mean Present 
1 Present 
  

Representative confocal image to confirm brain gene editing in female Ai9 mouse (mouse #C2B) with FUS array. (A) 10x whole section confocal image (section C2B5-5-2) to confirm Tomato activation only at the FUS-targeted site but not at the control site. The 1,200 um2 region of interest (ROI) with 20x magnification at (B) FUS-targeted and (C) control sites for neuron editing efficiency determination 


Publication Title
Focused ultrasound-mediated brain genome editing. NCBI
Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing. NCBI