Idiopathic Nephrotic Syndrome (INS) accounts for 85% of glomerular nephropathies in children and 25-30% of those in adults. Pathogenesis of this syndrome is based on an immune disorder whose mechanism remains incompletely understood. The team has identified a promising protein target to develop gene therapy for INS. However, no simple technique compatible with a gene therapy treatment has shown efficiency to target the podocyte so far. Vectors classically used in gene therapy (adenovirus, retrovirus, etc.) do not allow for proper targeting of the podocyte due to the presence of a glomerular barrier. Thus, the inventors have developed nanocapsules consisting of multilayers of chitosan/DNA assembly as a vector carrier allowing specific expression of miRNA in podocytes. This therapeutic approach has been validated in vitro and optimized for in vivo use, making it possible to envisage new therapeutic perspectives for INS in humans. This new innovative expression nanocapsule can be optimized for other therapeutic uses.
Keywords: Nanoparticules, DNA delivery, Non-viral carrier, Nephrotic syndrome, miRNA