Antisense strategies: delivery of oligonucleoides by nonviral nanoparticles

Claude Malvy (1) , Christine Vauthier (2)

1 UMR 8203 - Vectorologie et thérapeutiques anti-cancéreuses [Villejuif]
2 IGPS - Institut Galien Paris-Saclay

Gene silencing approaches have demonstrated high promise for the development of new treatments of diseases caused by the abnormal overexpression of a known gene. However, the in vivo delivery of the gene silencing mediating agents (antisense oligonucleotides and siRNA) is a bottleneck for the success of these innovative treatments. Solutions for their delivery are emerging from the nanomedicines thanks to the extremely active research aiming to find suitable delivery methods. This paper summarizes the main characteristics in term of their composition and structure of the most advanced carriers designed for siRNA and antisense oligonucleotide delivery. It also summarizes the results of the activity produced in vivo by several of these systems. The conclusion highlights open questions and bottlenecks that remain on the way to clinical development at a large scale.

Domaines: Pharmacie galénique

Claude Malvy, Christine Vauthier. Antisense strategies: delivery of oligonucleoides by nonviral nanoparticles. Joshua D Ramsey; M Laird Forrest. Nanoparticles for Biotherapeutic Delivery, 1, Future Science Ltd, pp.6-21, 2015, 978-1-910420-40-9. ⟨10.4155/fseb2013.14.142⟩. ⟨hal-03196151⟩