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ExperimentIndependent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Experiment Conditioncc47-SaCas9, Female
Assay Description: Editing efficiency calculated by counting fluorescent cells (RFP) compared to DAPI stained nuclei
Tissue Measured:heart
Cell Type :cardiac muscle cell
Measurement Type:Editing Efficiency
Record ID15000001270

EditorSauCas9
Delivery System
Delivery System Subtype
GuideAi9 SaCas9 Guide A; Ai9 SaCas9 Guide B
VectorAAVcc47-SaCas9-Ai9;

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

Application Methodintravenous (tail vein)
Application Siteliver and heart
Dosage1.0x10^14 vg copies/kg (vg/kg) (2.0x10^12/20g mouse)
Injection Frequencyonce
Injection Rate
Injection Volume200 microliters
Days post injection4 weeks
Editor FormatAAV vector encoded
Antidote Id
Antidote Description

Measured Values
Samples Size: 5
 
Replicate Result (Editing Efficiency)
Measurement Units: % of cells
Mean 4.83 
1 4.66 
2 5.86 
3 2.9 
4 5.11 
5 5.61 
  

Title Description File Download SCGE ID
Heaney_SATC Tissue Processing, Imaging and Analysis Procedure for tissue preparation, imaging and analysis. Heaney_SATC_Tissue_Processing_Imaging_and_Analysis.pdf 21000000025