Validation Experiment: Independent validation of Chaikof delivery platform using virus-like particle (VLP) delivery to the mouse liver
PI: Jason D Heaney, PhD
Description: Virus-like particles carrying a CRISPR/Cas base editor and a guide RNA targeting the PSCK9 locus were injected i.v. into male and female mice. One week after injection, the mice were dissected, and genomic DNA isolated from a panel of organs. Targeted NGS was performed to evaluate the degree of editing at the PCSK9 locus in the liver (primary target), and non-target organs. Two potential off-target editing sites (OT6 and OT7) were also sequenced.
Editing Assay:
Targeted next generation sequencing (NGS)
Parent Project: BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Chaikof Validation (Chaikof, Elliot L. )
Download: Submitted files
Original Experiment/s that are being validated:
Organ System Overview
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