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

ERGANEO



26 Mars 2021

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Fields

Biology / Medical

Sectors

Chemicals, Materials & Plant-based Materials

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.

Applications:

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

Competitive advantages:

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

Keywords: Nanoparticules, DNA delivery, Non-viral carrier, Nephrotic syndrome, miRNA

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