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Experiment: Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.

PI:   Aravind Asokan, PhD 

A dual vector strategy was employed: one self complementary vector delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (single stranded vector). This strategy was evaluted with both AAV9 (n=4) and AAVcc47 (n=4) by intravenous injection in Ai9 mice. A total dose of 4e12vg was injected into each mouse and vectors mixed in a 1:1 ratio of cas9 to guide RNA (2e12vg of CMV Sacas9 vector and 2e12vg of the self complementary sgRNA vector) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart.
Editing Assay: - Editing efficiency calculated by counting fluorescent cells (TdTomato) compared to DAPI stained cells.

Organ System Overview

AAV9, Male
AAV9, Female
AAVcc47, Male
AAVcc47, Female
Delivery Efficiency
Editing Efficiency
Target Tissue
Not Available

Analyze Data Sets Available for this Experiment
Liver and Biliary
  Liver (unspecified)
  Heart (unspecified)