RT info:eu-repo/semantics/article
T1 Amphiphilic Cationic Carbosilane-PEG Dendrimers: Synthesis and Applications in Gene Therapy
A1 Sánchez-Nieves Fernández, Javier
A1 Fransen, Peter
A1 Pulido, Daniel
A1 Llorente Heras, Raquel
A1 Muñoz Fernández, María Ángeles
A1 Albericio, Fernando
A1 Royo, Miriam
A1 Gómez Ramírez, Rafael
A1 Mata de la Mata, Francisco Javier de la
K1 Dendrimers
K1 Click chemistry
K1 Carbosilane
K1 PEG
K1 Gene therapy
K1 HIV
K1 Química
K1 Chemistry
AB Here we synthesized carbosilane, generation 1 to 3, and PEG-based dendrons functionalized at the periphery with NHBoc groups and at the focal point with azide and alkyne moieties, respectively. The coupling of these two types of dendrons via click chemistry led to the formation of new hybrid dendrimers with two distinct moieties, the hydrophobic carbosilane and the hydrophilic PEG-based dendron. The protected dendrimers were transformed into cationic ammonium dendrimers. These unique amphiphilic dendrimers were studied as vectors for gene therapy against HIV in peripheral blood mononuclear cells (PBMC) and their performance was compared with that of a PEG-free carbosilane dendrimer. The presence of the PEG moiety afforded lower toxicities and evidenced a weaker interaction between dendrimers and siRNA when compared to the homodendrimer analogous. Both features, lower toxicity and lower dendriplex strength, are key properties for use of these vectors as carriers of nucleic material.
PB Elsevier
SN 0223-5234
YR 2014
FD 2014-01-31
LK http://hdl.handle.net/10017/33945
UL http://hdl.handle.net/10017/33945
LA eng
NO Comunidad de Madrid
DS MINDS@UW
RD 24-abr-2024