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Project: Efficient In Vivo RNP-Based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles

PI:   Zheng-Yi Chen, PhD NIH Report

The proposal is designed to screen lipid nanoparticles as novel materials for RNP (ribonucleoprotein) delivery of editing machinery into the mammalian sensory organ inner ear, to expand the cell types that can be edited to treat genetic hearing loss and to establish a method to perform the study in wildtype large animals. This study has direct relevance to bringing editing based therapy to clinic.
Summary of data submissions:
  • Data for 3  experiments were submitted on 2021-04-13 SCGE ID:1029
  • 1 of 3 experiments have been validated

Submissions Details

SCGE ID:1029 - Submission Date: 2021-04-13 
Experiment Name Type Description
Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in primary fibroblast cells from adult Ai14 mouse cochlea

In Vitro LNP delivery of Cas9 mRNA/sgRNA in primary fibroblast cells from adult Ai14 mouse cochlea (inner ear)
Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice

In Vivo Delivery of firefly luciferase mRNA via new Lipid NanoParticles by tail vein injection into WT C57BL/6J mice targeting the Liver and delivery is measured by luciferase expression.
Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea

In Vivo Delivery of Cas9/sgRNA mRNA via new LNPs to the cochlea by cochleostomy and gene editing is measured by percentage of tdTomato positive cells.