The lab of Youyang Zhao, PhD, of the Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital in Chicago has developed a unique nanoparticle to deliver genome editing technology, including CRISPR/Cas9, to endothelial cells, cells that make up the bloodline. . vessel walls. This is the first time vascular endothelial cells can be reached for genome editing, as the usual way of delivering CRISPR/Cas9 – via a virus – does not work for this cell type. The findings were published in the journal Cell Reports.
The nanoparticle we have developed is a powerful new delivery system for genome editing in vascular endothelial cells and could be used to treat many diseases, including the acute respiratory distress syndrome of severe COVID-19. With this nanoparticle we can introduce genes to inhibit vascular damage 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 simultaneously. This is an important advance for the treatment of diseases caused by endothelial dysfunction.”
dr. Youyang Zhao, senior author, 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 in endothelial cells could even treat cancers by cutting off the blood supply to the tumor or blocking cancer metastases.
At this stage, Dr. Zhao and colleagues provided excellent results in a mouse model. The nanoparticle carrying CRISPR/Cas9 plasmid DNA was introduced via a single IV injection and took a few days to be effective. Preclinical testing will be needed before clinical trials can begin.
“Our nanoparticle delivery system for genome editing and transgene expression is also a huge advance for cardiovascular research,” added Dr. Zhao to it.
Ann & Robert H. Lurie Children’s Hospital in Chicago