All experiments with live animals were approved by the Institutional Animal Care and Use Committee (IACUC) of the Shanghai Institute of Nutrition and Health, Chinese Academy of Sciences. B6-EGFP mice (C57BL6-Tg (CAG-EGFP)10sb/J) were purchased from Jackson Laboratory. The male and female mice were evenly assigned to groups of predetermined sample size. AAV particles encoding AI-REWIRE 4.1 or CU-REWIRE 3.0 were injected into the B6-EGFP mice through tail intravenous injection (2×1012 vector genomes per mouse in 4-week-old B6-EGFP mice (5 (link),6 (link))). Four weeks after injection, we collected muscles and heart samples from B6-EGFP mice and evaluated corresponding editing efficiency via RNA-Seq and Sanger-seq. No visible symptoms, such as a rough hair coat and moved slowly were observed in all mice after injection.
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In Vivo Gene Editing Efficiency Assessment
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Corresponding Organization : Center for Excellence in Molecular Cell Science
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Variable analysis
independent variables
- AAV particles encoding AI-REWIRE 4.1 or CU-REWIRE 3.0
- Editing efficiency in muscles and heart samples, evaluated via RNA-Seq and Sanger-seq
- Male and female B6-EGFP mice were evenly assigned to groups of predetermined sample size
- No visible symptoms, such as a rough hair coat and moved slowly, were observed in all mice after injection
- Positive control: B6-EGFP mice (C57BL6-Tg (CAG-EGFP)10sb/J) purchased from Jackson Laboratory
- Negative control: Not explicitly mentioned
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