Polyurea dendrimer for efficient cytosolic siRNA delivery

• Autor(a) principal: Restani, Rita
• Data de Publicação: 2014
• Outros Autores: Conde, João, Baptista, Pedro, Cidade, Maria Teresa Varanda, Bragança, Ana, Morgado, Jorge, Correia, Ilídio Joaquim Sobreira, Ricardo, Ana Aguiar, Bonifácio, Vasco
• Tipo de documento: Artigo
• Idioma: eng
• Título da fonte: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
• Texto Completo: http://hdl.handle.net/10400.6/4663
• topic: SiRNA; Dendrimer; Polyurea

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.