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Experiment Record Detail

ExperimentTesting gRNA sequence and gRNA scaffold modified in Ai9 mice.
Experiment ConditionMale
Assay Description: Editing efficiency assessed by quantifying tdTomato positive area above threshold
Tissue Measured:cerebral cortex
Cell Type :astrocyte
Measurement Type:Editing Efficiency
Record ID15000000949
EditorSaCas9
Delivery System
Delivery System Subtype
GuideR2-modified; L1-modified
VectorBI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2; BI28:AAV-GFAP-SaCas9-WPRE3-pA;
Model SpeciesMouse
Model NameB6.Cg-Gt(ROSA)26Sor ^ tm9(CAG-tdTomato)Hze/J
Sample SexMale
Application Methodintravenous, retro-orbital
Application Sitecentral nervous system
Dosage3e11 vg each virus
Injection Frequencyonce
Injection Rate
Injection Volume50-100 microliters
Days post injection4 weeks
Editor FormatAAV vector encoded
Antidote Id
Antidote Description
Measured Values
Samples Size: 4
 
Replicate Result (Editing Efficiency)
Measurement Units: tdTomato positive area
Mean 38.7 
1 41.8 
2 60.1 
3 27.4 
4 25.5 
  

Experiment Wide Protocols

Title Description File Download SCGE ID
Deverman_Comprehensive Methods Procedure for plasmid cloning, editing evaluation in fibroblast, AAV production and administration, tissue processing and IHC. Deverman_Comprehensive_methods.pdf 21000000012
Deverman_Area Based Quantification of Editing Efficiency Protocol Procedure for non-IHC based image quantification of editing. Deverman_Area_based_quantification_of_editing_efficiency.pdf 21000000011

Associated Publications
Publication Title
Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing. NCBI