1. Inside out: optimization of lipid nanoparticle formulations for exterior complexation and in vivo delivery of saRNA. Blakney AK et al. 2019. Gene Therapy

Self-amplifying RNA (saRNA) therapies are at the forefront of new vaccine design, as they have been used to treat diseases like HIV or cancer.  To make their effect saRNA molecules must go through the membrane of the cells, but their biochemical properties make it difficult As nucleotidic molecules  they are very sensitive to enzymes that can degrade them. Thus, saRNA molecules need a delivery vehicle that allows them to reach their destination without losing its chemical integrity.

In this context, lipid nanoparticles (LNPs) have been used to encapsulate RNA-based therapeutic molecules. This article shows that LNPs can protect saRNAs successfully even when they are only adsorbed to their surface. This opens the door to a new strategy to deliver RNA-based therapies, like siRNA for IBD! To the publication > 

 2. gH625 cell-penetrating peptide promotes the endosomal escape of nanovectorized siRNA in a triple negative breast cancer cell line. Djemaa et al. 2019. Biomacromolecules.

Small interfering RNAs (siRNA) stand as a promising cancer therapy, but to make their effect they must cross the membrane of the tumorous cells. Thus, they need a delivery system to help them overcome this barrier, usually nanoparticles that encapsulate the siRNA molecules. A possible deliver strategy is to take advantage of the molecular uptake systems naturally present in cells. This can be done by adding cell-penetrating molecules to the surface of the delivery particles. 

In this article, researchers have used protein gH625 to enhance the trafficking of siRNA into the cells in a triple-negative breast cancer model. The uptake of the therapeutic RNA molecules was higher when the cell-penetrating protein was covering the surface of the nanocarrier.This strategy may be useful to treat other diseases with siRNA! To the publication >