Validation Experiment: Independent validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear
PI: Stephen A Murray, PhD
Description: 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.
Parent Project: The Jackson Laboratory Gene Editing Testing Center - Chen Validation (Chen, Zheng-Yi)
-
Other experiments in this project: 1
Download: Submitted files
Original Experiment/s that are being validated:
Results |
| Editing Efficiency | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Condition | Tissue | Cell Type | Sex | Age | Editor | Model | Delivery | Target Locus | Guide | Injection Frequency | Signal | Image |
| Arm 1: 306-O12B LNP containing Cas9 mRNA and sgRNA | base of cochlear canal (TARGET) | Mixed | 8 weeks | CleanCap® Cas9 mRNA (5moU) | Ai14 mouse (congenic) | 306-O12B | Ai9/Ai14 reporter transgene | sg298 | once | |||
| Arm 1: 306-O12B LNP containing Cas9 mRNA and sgRNA | scala tympani (TARGET) | Mixed | 8 weeks | CleanCap® Cas9 mRNA (5moU) | Ai14 mouse (congenic) | 306-O12B | Ai9/Ai14 reporter transgene | sg298 | once | |||
| Arm 2: 306-S10 LNP containing Cas9 mRNA and gRNA | base of cochlear canal (TARGET) | Mixed | 8 weeks | CleanCap® Cas9 mRNA (5moU) | Ai14 mouse (congenic) | 306-S10 | Ai9/Ai14 reporter transgene | sg298 | once | |||
| Arm 2: 306-S10 LNP containing Cas9 mRNA and gRNA | scala tympani (TARGET) | Mixed | 8 weeks | CleanCap® Cas9 mRNA (5moU) | Ai14 mouse (congenic) | 306-S10 | Ai9/Ai14 reporter transgene | sg298 | once | |||
| Arm 3: uninjected control | base of cochlear canal (TARGET) | Mixed | 8 weeks | Ai14 mouse (congenic) | none | |||||||
| Arm 3: uninjected control | scala tympani (TARGET) | Mixed | 8 weeks | Ai14 mouse (congenic) | none | |||||||
![]() |
![]() |

