CRISPR/Cas9 Delivery Mediated with Hydroxyl-Rich Nanosystems for Gene Editing in Aorta.

ABSTRACT: A CRISPR/Cas9 system has emerged as a powerful tool for gene editing to treat genetic mutation related diseases. Due to the complete endothelial barrier, effective delivery of the CRISPR/Cas9 system to vasculatures remains a challenge for in vivo gene editing of genetic vascular diseases especially in aorta. Herein, it is reported that CHO-PGEA (cholesterol (CHO)-terminated ethanolamine-aminated poly(glycidyl methacrylate)) with rich hydroxyl groups can deliver a plasmid based pCas9-sgFbn1 system for the knockout of exon 10 in Fbn1 gene. This is the first report of a polycation-mediated CRISPR/Cas9 system for gene editing in aorta of adult mice. CHO-PGEA/pCas9-sgFbn1 nanosystems can effectively contribute to the knockout of exon 10 in Fbn1 in vascular smooth mus

SUBMITTER: Zhang X 

PROVIDER: S-EPMC6662060 | biostudies-literature | 2019 Jun

REPOSITORIES: biostudies-literature