Health
Unique nanoparticles can provide genomic editing technology to endothelial cells
Ann & Robert H. Lully Children’s Hospital of Chicago’s Stanley Man Children’s Research Institute, Youyang Zhao, PhD’s lab, is working to deliver genome-editing technology, including CRISPR / Cas9, to endothelial cells, the cells that line blood. Developed a unique nanoparticle of blood vessel wall. This is the first time that vascular endothelial cells can be reached for genome editing, as the usual method of delivering CRISPR / Cas9 via virus does not work for this cell type.The survey results were published in the journal Cell report..
The nanoparticles we have developed are powerful new delivery systems for genome editing of vascular endothelial cells and can be used to treat many diseases, including acute respiratory distress syndrome due to severe COVID-19. These nanoparticles can be used to introduce genes that suppress vascular damage, promote vascular repair, correct gene mutations, and turn genes on or off to restore normal function. You can also edit multiple genes at the same time. This is an important advance in treating any disease caused by endothelial dysfunction. “
Dr. Youyang Zhao, Senior Author of Lurie Children’s
Endothelial dysfunction is at the root of many diseases such as coronary artery disease, stroke, bronchopulmonary dysplasia, and pulmonary arterial hypertension. Dr. Zhao explained that genome editing of endothelial cells can even cure cancer by blocking the blood supply to the tumor or blocking the metastasis of the cancer.
At this stage, Dr. Zhao and colleagues achieved excellent results with the mouse model. Nanoparticles carrying CRISPR / Cas9 plasmid DNA were introduced by a single IV injection and took several days to be effective. Preclinical trials are required before starting a clinical trial.
“Our nanoparticle delivery system for genome editing and transgene expression is also a major advance for cardiovascular research,” Dr. Zhao added.
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