Cationic Carbosilane Dendrimers as Non‐viral Vectors of Nucleic Acids (Oligonucleotide or siRNA) for Gene Therapy Purposes

A new approach to develop novel non‐viral vector for HIV inhibition based on carbosilane dendrimers is described. Cationic ammonium‐terminated carbosilane dendrimers have been synthesized via alcoholysis of dendritic Si‐Cl bonds or hydrosylilation reactions with dendritic Si‐H bonds. Ammonium‐terminated carbosilane dendrimers are able to form dendriplexes which showed good biocompatibility profiles in vitro, and protect nucleic acids from binding to serum proteins or RNases and degradation. Finally, they were found to be a successful and efficient vehicle for the transport and delivery of genetic material (ODN or siRNA) to a wide range of immune cells, and according to this, a specific reduction of HIV p24 antigen release by anti‐HIV ODN or siRNA was observed.

Therefore, the carbosilane dendrimers presented here are in general good candidates as non‐viral vectors in the context of biological applications of small nucleic acids, and in particular, they may present a new interesting approach for the treatment of HIV infection.