Scientists develop “a powerful new delivery system for genome editing in vascular endothelial cells, and could be used to treat many diseases” including SARS-COV-2.
A team of scientists has discovered a novel nanoparticle that can carry genome editing technology, such as CRISPR/Cas9, to endothelial cells, which are the cells that line the walls of blood vessels, in a targeted manner.
This is the first time that vascular endothelial cells could be targetted for genome editing, since the usual way to deliver CRISPR/Cas9 – through a virus – does not work for this cell type.
The results of the study 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,” says senior author Dr. Zhao from Lurie Children’s.
“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.”
In many disorders, such as coronary artery disease and stroke but also bronchopulmonary dysplasia and pulmonary artery hypertension, endothelial dysfunction lies at the base of the problem.
As Dr. Zhao noted, genome editing in endothelial cells may even be used to cure cancer by cutting off the blood supply to the tumor or by inhibiting cancer metastasis.
Dr. Zhao and colleagues had already produced outstanding results in a mouse model at this point.
The nanoparticle-containing CRISPR/Cas9 plasmid DNA was introduced into the body by intravenous injection and took several days to become effective.
Preclinical testing will be required prior to the start of clinical trials.
“Our nanoparticle delivery system for genome editing and transgene expression also is a huge advance for cardiovascular research,” adds Dr. Zhao.
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