Novel delivery system for genome editing in vascular endothelial cells developed

The laboratory of Youyang Zhao, PhD, of the Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago, developed a novel nanoparticle to deliver genome editing technology, such as CRISPR/Cas9, to endothelial cells, which line blood vessel walls.

Genome Editing

Image Credit: Natali _ Mis/Shutterstock.com

Since the usual method of delivering CRISPR/Cas9—via a virus—does not work for this cell type, this is the first time vascular endothelial cells have been reached for genome editing. The research findings were published in the journal Cell Reports.

The nanoparticle we developed is a powerful new delivery system for genome editing in vascular endothelial cells, and could be used to treat many diseases, including acute respiratory distress syndrome from severe COVID-19.

Youyang Zhao, Senior Author, Ann & Robert H. Lurie Children’s Hospital of Chicago

Dr Zhao adds, “With this nanoparticle we can introduce genes to inhibit vascular injury and/or promote vascular repair, correct gene mutations, and turn genes on or off to restore normal function. It also allows us to edit multiple genes at the same time. This is an important advance for treating any disease caused by endothelial dysfunction.”

Many diseases, including coronary artery disease, stroke, bronchopulmonary dysplasia, and pulmonary artery hypertension, are caused by endothelial dysfunction. Dr. Zhao clarified that genome editing in endothelial cells could also be utilized to help fight cancer by cutting off the tumor’s blood supply or preventing cancer metastasis.

Dr Zhao and colleagues achieved great success in a mouse model. The nanoparticle containing the CRISPR/Cas9 plasmid DNA was introduced via a one-time IV injection and took a few days to become potent. Before clinical trials can begin, preclinical testing will be required.

Our nanoparticle delivery system for genome editing and transgene expression also is a huge advance for cardiovascular research.”

Youyang Zhao, Senior Author, Ann & Robert H. Lurie Children’s Hospital of Chicago

Source:
Journal reference:

Zhang, X., et al. (2022) Robust genome editing in adult vascular endothelium by nanoparticle delivery of CRISPR-Cas9 plasmid DNA. Cell Reports. doi.org/10.1016/j.celrep.2021.110196.

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