aptamer-VEGF-A saRNA chimera

The aptamer-VEGF-A saRNA chimera is an experimental, preclinical RNA-based therapeutic designed to improve graft survival and revascularization in pancreatic islet transplantation for diabetes. The chimera consists of a beta-cell-targeting RNA aptamer (such as 1-717 or m12-3773) conjugated to a species-specific small activating RNA (saRNA) targeting the vascular endothelial growth factor A (VEGF-A) promoter. By binding to cell-surface proteins on pancreatic beta-cells (such as TMED6 or clusterin), the aptamer delivers the saRNA payload directly into the cells. Once inside, the saRNA activates the endogenous VEGF-A gene locus in an Argonaute-2-dependent manner, driving transient, stress-independent upregulation of VEGF-A. This localized expression of VEGF-A accelerates host-derived revascularization and perfusion of the transplanted islets, reducing post-transplant autophagic stress and improving functional glucose control without requiring permanent genomic modification.

Other names

aptamer-VEGF-A small activating RNA chimerabeta-cell-targeted aptamer-VEGF-A saRNA chimerasaRNAVEGF-A aptamer chimera