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ExperimentIndependent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Experiment ConditionArm1a: BI28 Neuron Specific Promoter, Experimental Animals, Males
Assay Description: Qualitative assesment of prevalence of tdTomato-positvie cells throughout the entire brain
Tissue Measured:brain
Cell Type :neuron
Measurement Type:Editing Efficiency
Record ID15000001448

EditorSaCas9
Delivery System
Delivery System Subtype
GuideL1-modified; L2-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

Application Method
Application Sitebrain
Dosage0.25E+11 viral genomes/gram for each virus
Injection Frequencyonce
Injection Rate
Injection Volume5 microliters/gram
Days post injection4 weeks
Editor FormatAAV vector encoded
Antidote Id
Antidote Description

Measured Values
Samples Size: 5
 
Replicate Result (Editing Efficiency)
Measurement Units: Signal
Mean 5 of 5 present 
1 Present 
2 Present 
3 Present 
4 Present 
5 Present 
  

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

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