NEW NANOPARTICLES WITH TARGETED EXPRESSION VECTOR AS A THERAPEUTIC APPROACH FOR IDIOPATHIC NEPHROTIC SYNDROME

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.

Competitive advantages

• Adaptable physico-chemical properties of nanocapsules
• Protection of miRNA against nucleases
• Production of active miRNA specifically in podocytes

Applications 

• Targeting of miRNA as a new therapeutic approach for Nephrotic syndrome
• Specific DNA expression vector targeted to the podocytes
• miRNA therapeutic approaches

Intellectual property 

WO2018141865A1

Key words

Nanoparticules - DNA delivery - Non-viral carrier - Nephrotic syndrome - miRNA