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Experiment: Second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear

PI:   Stephen A Murray, PhD 

Delivery of CRISPR/Cas9 via bioreducible lipid nanoparticles (LNPs) to the inner ear in Ai14 mice
Editing Assay: - Confocal Imaging of mouse cochlea, direct fluorescence RFP (tdTomato) compared to Hoechst stained nuclei.

Organ System Overview
Delivery Efficiency
Editing Efficiency
Target Tissue
Not Studied

Arm 3: uninjected control
Arm 1: 306-O12B LNP containing Cas9 mRNA and sgRNA
Arm 2: 306-S10 LNP containing Cas9 mRNA and gRNA

Analyze Data Sets Available for this ExperimentDeliveryEditing
  Base of cochlear canal  (TARGET) n/a
  Scala tympani  (TARGET) n/a