• Cidade, Maria Teresa
• Morgado, Jorge
• Conde, João
• Baptista, Pedro V.
• Bonifácio, Vasco D. B.
• Aguiar-Ricardo, Ana
• Bragança, Ana M.
• Restani, Rita B.
• Correia, Ilídio J.

Abstract

The design of small interfering RNA (siRNA) delivery materials showing efficacy in vivo is at the forefront of nanotherapeutics research. Polyurea (PURE-type) dendrimers are ‘smart’ biocompatible 3D polymers that unveil a dynamic and elegant back-folding mechanism involving hydrogen bonding between primary amines at the surface and tertiary amines and ureas at the core. Similarly, to a biological proton pump, they are able to automatically and reversibly transform their conformation in response to pH stimulus. Here, we show that PURE-G4 is a useful gene silencing platform showing no cellular toxicity. As a proof of concept we investigated the PURE-G4-siRNA dendriplex, which was shown to be an attractive platform with high transfection efficacy. The simplicity associated with the complexation of siRNA with polyurea dendrimers makes them a powerful tool for efficient cytosolic siRNA delivery. ; We acknowledge LabRMN at FCT/UNL and Rede Nacional de RMN for access to the facilities. Rede Nacional is supported with funds from Fundação para a Ciência e a Tecnologia (FC&T, Lisbon), Projecto de Re-equipamento Científico, Portugal. We thank the financial support from FC&T through Strategic Projects PEst-C/CTM/LA0025/2013, PEst-OE/SAU/UI0009/2013 and PEst-C/EQB/LA0006/2013 (FC&T: financial support to CENIMAT/I3N, CIGMH and REQUIMTE) and projects PTDC/EQU-EQU/116097/2009, PTDC/BBB-NAN/1812/2012, PTDC/CTM/099452/2008, PhD grants SFRH/BD/66858/2009 (R.B.R.) and SFRH/BD/62957/2009 (J.C.). ; Peer reviewed

Topics

• impedance spectroscopy
• surface
• polymer
• Hydrogen
• toxicity
• amine
• dendrimer