Sjögren’s syndrome is a systemic chronic autoimmune inflammatory disease that primarily targets the salivary and lacrimal glands (LGs). Currently there is no cure; therefore, cell-based regenerative therapy may be a viable option. LG inflammation is facilitated by extracellular ATP and mediated by the Pannexin-1 (Panx1) membrane channel glycoprotein. We propose that suppression of inflammation through manipulation of Panx1 activity can stimulate epithelial cell progenitor (EPCP) engraftment.
RNA interference (RNAi) is a natural mechanism for the modulation of gene expression by small interfering RNAs (siRNAs). A broad range of human diseases, including cancer, metabolic disorders, and neurodegeneration can be treated via the silencing of specific genes using siRNAs. Early attempts to harness RNAi for therapeutic development focused on lipid- or nanoparticle-formulated, minimally modified siRNAs (reviewed in (1,2)). Recently, non-formulated, conjugate-mediated delivery emerged as an alternative, clinically dominant delivery paradigm. By changing the nature of the ligand, this approach has the potential to enable targeted delivery to a variety of tissues.
