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ExperimentIndependent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Experiment ConditionArm 2a: BI28 Astrocyte Specific Promoter, Experimental Animals, Males
Assay Description: Quantitative assessment of tdTomato-positive cells per DAPI-stained nucleus in a specific brain region
Tissue Measured:dorsal plus ventral thalamus
Cell Type :astrocyte
Measurement Type:Editing Efficiency
Record ID15000001455

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: 6
 
Replicate Result (Editing Efficiency)
Measurement Units: % Area
Mean 43.4 
1 53.5 
2 29.5 
3 29.2 
4 53.8 
5 46.4 
6 48.0 
  

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