Polyurea dendrimer for efficient cytosolic siRNA delivery

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...

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Detalhes bibliográficos
Autor principal: Restani, Rita (author)
Outros Autores: Conde, João (author), Baptista, Pedro (author), Cidade, Maria Teresa Varanda (author), Bragança, Ana (author), Morgado, Jorge (author), Correia, Ilídio Joaquim Sobreira (author), Ricardo, Ana Isabel Nobre Martins Aguiar de Oliveira (author), Bonifácio, Vasco (author)
Formato: article
Idioma:eng
Publicado em: 2018
Assuntos:
Texto completo:http://hdl.handle.net/10400.6/4663
País:Portugal
Oai:oai:ubibliorum.ubi.pt:10400.6/4663
Descrição
Resumo: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.