In vivo targeted gene delivery to peripheral neurons

What is it about?

In this study we report the use of a non-viral neurotropic poly(ethylene imine)-based nanoparticle that is capable of mediating neuron-specific transfection upon a subcutaneous injection. Nanoparticles were targeted to peripheral neurons by using the non-toxic carboxylic fragment of tetanus toxin (HC), which besides neurotropic is capable of being retrogradely transported from neuron terminals to the cell bodies. The peripheral subcutaneous delivery of targeted nanoparticles resulted in the transfection of 56% and 64% of L4 and L5 dorsal root ganglia neurons, respectively, while spatially limited the transgene expression to non-neuronal tissues, in comparison to the controls.

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Why is it important?

Despite the wide number of non-viral gene delivery vectors proposed in the past, to the best of our knowledge, none has been developed to actively target peripheral neurons following a minimally-invasive peripheral route of administration. Thus, such development is of significant interest not only for safety issues, but also to aid in the design of more clinically relevant therapeutic applications to treat neuropathic conditions.