This therapy specifically targets inflammatory and vascular smooth muscle cells, which are key players in plaque buildup, while carefully preserving healthy endothelial cells. Initially, we employed an adenoviral vector, but our current focus is on a more advanced synthetic mRNA-based miRNA switch technology encapsulated in nanoparticles.

This innovative approach effectively delivers therapeutic payloads directly to damaged vessel areas, significantly reducing plaque formation and promoting vessel repair. We are actively enhancing this technology by combining it with siRNA to further suppress inflammation in an effort to restore the endothelial barrier while inhibiting the progression of atherosclerotic plaque.

The first use of CRISPR technology to transcriptionally activate the C19MC, a primate-specific microRNA cluster located on chromosome 19, led to a groundbreaking discovery. This research revealed a compelling example of convergent evolution between the C19MC and the rodent-specific microRNA cluster C2MC on chromosome 2. These clusters are primarily expressed in trophoblasts and contain short interspersed nuclear elements (SINEs), such as Alu (primate) or B1 (rodent) repeats.

The study demonstrated that the SINEs of these clusters produce double-stranded RNA (dsRNA), triggering a potent immune response similar to viral mimicry. This immune activation involves type III interferon signaling, offering essential antiviral protection to the fetus during development. Additionally, the research highlighted the crucial role of C19MC in regulating epithelial-to-mesenchymal transition and accelerating cell reprogramming processes.