Delivering siRNA with Dendrimers: In Vivo Applications
2017
Background: 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.
Material & Methods: 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.
Conclusion: 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 applications.
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