Over the last decades, gene therapy has emerged as a pioneering therapeutic approach to
treat or prevent several diseases. Among the explored strategies, the short-term silencing of protein
coding genes mediated by siRNAs has a good therapeutic potential in a clinical setting.
However, the widespread use of siRNA will require the development of clinically suitable, safe and
effective vehicles with the ability to complex and deliver siRNA into target cells with minimal toxicity.
Lately, dendrimers have gained considerable attention as non-viral vectors in nucleic acid delivery
due to their unique structural characteristics (globular, well defined and highly branched structure,
multivalency, low polydispersity and tunable nanosize), along with their relevant capacity to complex
and protect nucleic acids in compact nanostructures, which can be functionalized with targeting moieties
in order to get cell specificity.
Here, we present an overview of the state-of-the-art of the most significant and recent advances on the
use of dendrimers as siRNA delivery vectors, with particular focus on the in vivo applications. We will
cover the use of different dendrimers, distinct administration routes, toxicity issues, as well as the target
tissue or disease, highlighting the potential of dendrimers as nanocarriers for therapeutic and biomedical